{"content":"<div class=\"filter_values\"><\/div><div class=\"tab-content clearfix\"><div class=\"tab-pane active\" id=\"scientific-articles\"><div class=\"curate-inner-subtitle\">110 resources available:<\/div><div class=\"evidence_info_content ev_ed\" >\n\t\t\t\t<a class=\"info_title collapsed\"><\/a>\n<div class=\"\n-evidence info_title\" data-id=\"98494\"><a href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/multicenter-open-label-extension-study-intrathecal-heparan-n-sulfatase-patients-sanfilippo-syndrome-type-a\/\"><h2>A Multicenter Open-Label Extension Study of Intrathecal Heparan-N-Sulfatase in Patients With Sanfilippo Syndrome Type A<\/h2><\/a>\n<span class=\"info_title_sub_text\">Molecular genetics and metabolism<\/span><\/div>\n<div class=\"info_cont_hide\">Sanfilippo syndrome type A (mucopolysaccharidosis type IIIA) is a rare autosomal recessive lysosomal disorder characterized by deficient heparan-N-sulfatase (HNS) activity, and subsequent accumulation of heparan sulfate, especially in the central nervous system. The disease is associated with progressive neurodegeneration in early childhood. For this open-label extension study of a phase 2b clinical trial, we report on safety and cognitive declin...<div class=\"evidence-share-row\"><div class=\"clinical_share \"><div class=\"clinical_share news-bind\">\n    \t<div class=\"page-myBinder share-binder share-item\" data-tag=\"news-meeting\" data-id=\"98494\"><img src=\"https:\/\/www.onempsvoice.com\/application\/themes\/onevoice-child\/images\/bookmark.png\" style=\"width:20px;\">  Bookmark<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><div class=\"social-share-box\">\n                                                        <div class=\"social-share\"> <span class=\"icon-share\"><\/span> Share\n                                                        <div class=\"social-share-hov social-share-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"share-discuss share-item share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a href=\"\" class=\"page-discuss-courage\"><span class=\"icon-talk icon-left\"><\/span> Discuss in Social Wall<\/a>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"post-facebook courage-social-cont share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a  href=\"javascript:void(0)\" title=\"share on Facebook\" data-detailurl=\"https:\/\/www.onempsvoice.com\/rcuratenew\/multicenter-open-label-extension-study-intrathecal-heparan-n-sulfatase-patients-sanfilippo-syndrome-type-a\/\" class=\"btnfbShare\"><div  class=\"head_fb header_facebook_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-facebook\"><\/i> <\/div>Facebook<\/a> \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t  <div class=\"post-twitter courage-social-cont share-item-inner\">\n\t\t\t\t\t\t  <a data-href=\"https:\/\/twitter.com\/intent\/tweet?source=&text=:%20\" title=\"share on Twitter\" onclick=\"window.open('https:\/\/twitter.com\/intent\/tweet?text='+ encodeURIComponent('A Multicenter Open-Label Extension Study of Intrathecal Heparan-N-Sulfatase in Patients With Sanfilippo Syndrome Type A') + ' on @oneMPSvoice ' + encodeURIComponent('')); return false;\"><div  class=\"head_tweet header_twitter_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-twitter\"><\/i>  <\/div>Twitter<\/a>\n\t\t\t\t\t\t  <\/div><div class=\"share-team share-item share_with_rareteam share-item-inner\" post=\"98494\" ><span class=\"share-team-icon icon-left\"><\/span>Share to rareTeam<\/div>\n                                                        <\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><\/div><div class=\"trial_readmore\"><a class=\"button_grey\"  data-id=\"98494\" href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/multicenter-open-label-extension-study-intrathecal-heparan-n-sulfatase-patients-sanfilippo-syndrome-type-a\/\"  >\nread more\n<\/a><\/div>\n\n\n<\/div><\/div><div class=\"evidence_info_content ev_ed\" >\n\t\t\t\t<a class=\"info_title collapsed\"><\/a>\n<div class=\"\n-evidence info_title\" data-id=\"98496\"><a href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/impact-excluding-nonrandomized-studies-rare-diseases-efficacy-safety-enzyme-replacement-therapy-patients-mucopolysaccharidosis\/\"><h2>The Impact of Excluding Nonrandomized Studies From Systematic Reviews in Rare Diseases: &#8220;The Example of Meta-Analyses Evaluating the Efficacy and Safety of Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis&#8221;<\/h2><\/a>\n<span class=\"info_title_sub_text\">Frontiers in molecular biosciences<\/span><span class=\"info_title_sub_text\">Year: 2021<\/span><\/div>\n<div class=\"info_cont_hide\">Nonrandomized studies are usually excluded from systematic reviews. This could lead to loss of a considerable amount of information on rare diseases. In this article, we explore the impact of excluding nonrandomized studies on the generalizability of meta-analyses results on mucopolysaccharidosis (MPS) disease. A comprehensive search of systematic reviews on MPS patients up to May 2020 was carried out (CRD42020191217). The primary endpoint was th...<div class=\"evidence-share-row\"><div class=\"clinical_share \"><div class=\"clinical_share news-bind\">\n    \t<div class=\"page-myBinder share-binder share-item\" data-tag=\"news-meeting\" data-id=\"98496\"><img src=\"https:\/\/www.onempsvoice.com\/application\/themes\/onevoice-child\/images\/bookmark.png\" style=\"width:20px;\">  Bookmark<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><div class=\"social-share-box\">\n                                                        <div class=\"social-share\"> <span class=\"icon-share\"><\/span> Share\n                                                        <div class=\"social-share-hov social-share-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"share-discuss share-item share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a href=\"\" class=\"page-discuss-courage\"><span class=\"icon-talk icon-left\"><\/span> Discuss in Social Wall<\/a>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"post-facebook courage-social-cont share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a  href=\"javascript:void(0)\" title=\"share on Facebook\" data-detailurl=\"https:\/\/www.onempsvoice.com\/rcuratenew\/impact-excluding-nonrandomized-studies-rare-diseases-efficacy-safety-enzyme-replacement-therapy-patients-mucopolysaccharidosis\/\" class=\"btnfbShare\"><div  class=\"head_fb header_facebook_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-facebook\"><\/i> <\/div>Facebook<\/a> \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t  <div class=\"post-twitter courage-social-cont share-item-inner\">\n\t\t\t\t\t\t  <a data-href=\"https:\/\/twitter.com\/intent\/tweet?source=&text=:%20\" title=\"share on Twitter\" onclick=\"window.open('https:\/\/twitter.com\/intent\/tweet?text='+ encodeURIComponent('The Impact of Excluding Nonrandomized Studies From Systematic Reviews in Rare Diseases: &#8220;The Example of Meta-Analyses Evaluating the Efficacy and Safety of Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis&#8221;') + ' on @oneMPSvoice ' + encodeURIComponent('')); return false;\"><div  class=\"head_tweet header_twitter_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-twitter\"><\/i>  <\/div>Twitter<\/a>\n\t\t\t\t\t\t  <\/div><div class=\"share-team share-item share_with_rareteam share-item-inner\" post=\"98496\" ><span class=\"share-team-icon icon-left\"><\/span>Share to rareTeam<\/div>\n                                                        <\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><\/div><div class=\"trial_readmore\"><a class=\"button_grey\"  data-id=\"98496\" href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/impact-excluding-nonrandomized-studies-rare-diseases-efficacy-safety-enzyme-replacement-therapy-patients-mucopolysaccharidosis\/\"  >\nread more\n<\/a><\/div>\n\n\n<\/div><\/div><div class=\"evidence_info_content ev_ed\" >\n\t\t\t\t<a class=\"info_title collapsed\"><\/a>\n<div class=\"\n-evidence info_title\" data-id=\"98498\"><a href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/plasma-proteomic-analysis-morquio-disease\/\"><h2>Plasma Proteomic Analysis in Morquio A Disease<\/h2><\/a>\n<span class=\"info_title_sub_text\">International journal of molecular sciences<\/span><span class=\"info_title_sub_text\">Year: 2021<\/span><\/div>\n<div class=\"info_cont_hide\">Mucopolysaccharidosis type IVA (MPS IVA) is a lysosomal disease caused by mutations in the gene encoding the enzyme-acetylgalactosamine-6-sulfate sulfatase (GALNS), and is characterized by systemic skeletal dysplasia due to excessive storage of keratan sulfate (KS) and chondroitin-6-sulfate in chondrocytes. Although improvements in the activity of daily living and endurance tests have been achieved with enzyme replacement therapy (ERT) with recom...<div class=\"evidence-share-row\"><div class=\"clinical_share \"><div class=\"clinical_share news-bind\">\n    \t<div class=\"page-myBinder share-binder share-item\" data-tag=\"news-meeting\" data-id=\"98498\"><img src=\"https:\/\/www.onempsvoice.com\/application\/themes\/onevoice-child\/images\/bookmark.png\" style=\"width:20px;\">  Bookmark<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><div class=\"social-share-box\">\n                                                        <div class=\"social-share\"> <span class=\"icon-share\"><\/span> Share\n                                                        <div class=\"social-share-hov social-share-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"share-discuss share-item share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a href=\"\" class=\"page-discuss-courage\"><span class=\"icon-talk icon-left\"><\/span> Discuss in Social Wall<\/a>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"post-facebook courage-social-cont share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a  href=\"javascript:void(0)\" title=\"share on Facebook\" data-detailurl=\"https:\/\/www.onempsvoice.com\/rcuratenew\/plasma-proteomic-analysis-morquio-disease\/\" class=\"btnfbShare\"><div  class=\"head_fb header_facebook_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-facebook\"><\/i> <\/div>Facebook<\/a> \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t  <div class=\"post-twitter courage-social-cont share-item-inner\">\n\t\t\t\t\t\t  <a data-href=\"https:\/\/twitter.com\/intent\/tweet?source=&text=:%20\" title=\"share on Twitter\" onclick=\"window.open('https:\/\/twitter.com\/intent\/tweet?text='+ encodeURIComponent('Plasma Proteomic Analysis in Morquio A Disease') + ' on @oneMPSvoice ' + encodeURIComponent('')); return false;\"><div  class=\"head_tweet header_twitter_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-twitter\"><\/i>  <\/div>Twitter<\/a>\n\t\t\t\t\t\t  <\/div><div class=\"share-team share-item share_with_rareteam share-item-inner\" post=\"98498\" ><span class=\"share-team-icon icon-left\"><\/span>Share to rareTeam<\/div>\n                                                        <\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><\/div><div class=\"trial_readmore\"><a class=\"button_grey\"  data-id=\"98498\" href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/plasma-proteomic-analysis-morquio-disease\/\"  >\nread more\n<\/a><\/div>\n\n\n<\/div><\/div><div class=\"evidence_info_content ev_ed\" >\n\t\t\t\t<a class=\"info_title collapsed\"><\/a>\n<div class=\"\n-evidence info_title\" data-id=\"98499\"><a href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/detailed-pedigree-analyses-prenatal-diagnosis-family-mucopolysaccharidosis-type-ii\/\"><h2>Detailed Pedigree Analyses and Prenatal Diagnosis for a Family With Mucopolysaccharidosis Type II<\/h2><\/a>\n<span class=\"info_title_sub_text\">BMC medical genomics<\/span><span class=\"info_title_sub_text\">Year: 2021<\/span><\/div>\n<div class=\"info_cont_hide\">Mucopolysaccharidosis type II (MPS II) is an X-linked multisystem disorder caused by mutations in the gene encoding iduronate 2-sulfatase (IDS). The clinical manifestations of MPS II include skeletal deformities, airway obstruction, cardiomyopathy, and neurologic deterioration. MPS II has high genetic heterogeneity disorder, and\u2009~\u2009658 variants of IDS have been reported.<div class=\"evidence-share-row\"><div class=\"clinical_share \"><div class=\"clinical_share news-bind\">\n    \t<div class=\"page-myBinder share-binder share-item\" data-tag=\"news-meeting\" data-id=\"98499\"><img src=\"https:\/\/www.onempsvoice.com\/application\/themes\/onevoice-child\/images\/bookmark.png\" style=\"width:20px;\">  Bookmark<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><div class=\"social-share-box\">\n                                                        <div class=\"social-share\"> <span class=\"icon-share\"><\/span> Share\n                                                        <div class=\"social-share-hov social-share-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"share-discuss share-item share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a href=\"\" class=\"page-discuss-courage\"><span class=\"icon-talk icon-left\"><\/span> Discuss in Social Wall<\/a>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"post-facebook courage-social-cont share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a  href=\"javascript:void(0)\" title=\"share on Facebook\" data-detailurl=\"https:\/\/www.onempsvoice.com\/rcuratenew\/detailed-pedigree-analyses-prenatal-diagnosis-family-mucopolysaccharidosis-type-ii\/\" class=\"btnfbShare\"><div  class=\"head_fb header_facebook_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-facebook\"><\/i> <\/div>Facebook<\/a> \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t  <div class=\"post-twitter courage-social-cont share-item-inner\">\n\t\t\t\t\t\t  <a data-href=\"https:\/\/twitter.com\/intent\/tweet?source=&text=:%20\" title=\"share on Twitter\" onclick=\"window.open('https:\/\/twitter.com\/intent\/tweet?text='+ encodeURIComponent('Detailed Pedigree Analyses and Prenatal Diagnosis for a Family With Mucopolysaccharidosis Type II') + ' on @oneMPSvoice ' + encodeURIComponent('')); return false;\"><div  class=\"head_tweet header_twitter_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-twitter\"><\/i>  <\/div>Twitter<\/a>\n\t\t\t\t\t\t  <\/div><div class=\"share-team share-item share_with_rareteam share-item-inner\" post=\"98499\" ><span class=\"share-team-icon icon-left\"><\/span>Share to rareTeam<\/div>\n                                                        <\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><\/div><div class=\"trial_readmore\"><a class=\"button_grey\"  data-id=\"98499\" href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/detailed-pedigree-analyses-prenatal-diagnosis-family-mucopolysaccharidosis-type-ii\/\"  >\nread more\n<\/a><\/div>\n\n\n<\/div><\/div><div class=\"evidence_info_content ev_ed\" >\n\t\t\t\t<a class=\"info_title collapsed\"><\/a>\n<div class=\"\n-evidence info_title\" data-id=\"98500\"><a href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/inhibition-iduronic-acid-biosynthesis-ebselen-reduces-glycosaminoglycan-accumulation-mucopolysaccharidosis-type-i-fibroblasts\/\"><h2>Inhibition of Iduronic Acid Biosynthesis by Ebselen Reduces Glycosaminoglycan Accumulation in Mucopolysaccharidosis Type I Fibroblasts<\/h2><\/a>\n<span class=\"info_title_sub_text\">Glycobiology<\/span><span class=\"info_title_sub_text\">Year: 2021<\/span><\/div>\n<div class=\"info_cont_hide\">Mucopolysaccharidosis type I (MPS-I) is a rare lysosomal storage disorder caused by deficiency of the enzyme alpha-L-iduronidase, which removes iduronic acid in both chondroitin\/dermatan sulfate (CS\/DS) and heparan sulfate (HS) and thereby contributes to the catabolism of glycosaminoglycans (GAGs). To ameliorate this genetic defect, the patients are currently treated by enzyme replacement and bone marrow transplantation, which have a number of dr...<div class=\"evidence-share-row\"><div class=\"clinical_share \"><div class=\"clinical_share news-bind\">\n    \t<div class=\"page-myBinder share-binder share-item\" data-tag=\"news-meeting\" data-id=\"98500\"><img src=\"https:\/\/www.onempsvoice.com\/application\/themes\/onevoice-child\/images\/bookmark.png\" style=\"width:20px;\">  Bookmark<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><div class=\"social-share-box\">\n                                                        <div class=\"social-share\"> <span class=\"icon-share\"><\/span> Share\n                                                        <div class=\"social-share-hov social-share-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"share-discuss share-item share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a href=\"\" class=\"page-discuss-courage\"><span class=\"icon-talk icon-left\"><\/span> Discuss in Social Wall<\/a>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"post-facebook courage-social-cont share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a  href=\"javascript:void(0)\" title=\"share on Facebook\" data-detailurl=\"https:\/\/www.onempsvoice.com\/rcuratenew\/inhibition-iduronic-acid-biosynthesis-ebselen-reduces-glycosaminoglycan-accumulation-mucopolysaccharidosis-type-i-fibroblasts\/\" class=\"btnfbShare\"><div  class=\"head_fb header_facebook_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-facebook\"><\/i> <\/div>Facebook<\/a> \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t  <div class=\"post-twitter courage-social-cont share-item-inner\">\n\t\t\t\t\t\t  <a data-href=\"https:\/\/twitter.com\/intent\/tweet?source=&text=:%20\" title=\"share on Twitter\" onclick=\"window.open('https:\/\/twitter.com\/intent\/tweet?text='+ encodeURIComponent('Inhibition of Iduronic Acid Biosynthesis by Ebselen Reduces Glycosaminoglycan Accumulation in Mucopolysaccharidosis Type I Fibroblasts') + ' on @oneMPSvoice ' + encodeURIComponent('')); return false;\"><div  class=\"head_tweet header_twitter_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-twitter\"><\/i>  <\/div>Twitter<\/a>\n\t\t\t\t\t\t  <\/div><div class=\"share-team share-item share_with_rareteam share-item-inner\" post=\"98500\" ><span class=\"share-team-icon icon-left\"><\/span>Share to rareTeam<\/div>\n                                                        <\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><\/div><div class=\"trial_readmore\"><a class=\"button_grey\"  data-id=\"98500\" href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/inhibition-iduronic-acid-biosynthesis-ebselen-reduces-glycosaminoglycan-accumulation-mucopolysaccharidosis-type-i-fibroblasts\/\"  >\nread more\n<\/a><\/div>\n\n\n<\/div><\/div><div class=\"evidence_info_content ev_ed\" >\n\t\t\t\t<a class=\"info_title collapsed\"><\/a>\n<div class=\"\n-evidence info_title\" data-id=\"98511\"><a href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/open-label-phase-1-2-study-vestronidase-alfa-mucopolysaccharidosis-vii\/\"><h2>Open-Label Phase 1\/2 Study of Vestronidase Alfa for Mucopolysaccharidosis VII<\/h2><\/a>\n<span class=\"info_title_sub_text\">Molecular genetics and metabolism reports<\/span><span class=\"info_title_sub_text\">Year: 2021<\/span><\/div>\n<div class=\"info_cont_hide\">Vestronidase alfa is an enzyme replacement therapy for mucopolysaccharidosis VII (MPS VII). In this open-label, phase 1\/2 study, three subjects with MPS VII received intravenous vestronidase alfa administered every other week (QOW) for 14\u00a0weeks (2\u00a0mg\/kg), followed by 24-week forced-dose titration (1, 4, and 2\u00a0mg\/kg QOW; 8\u00a0weeks each), 36-week continuation (2\u00a0mg\/kg), and long-term extension (4\u00a0mg\/kg). Vestronidase alfa was well tolerated and...<div class=\"evidence-share-row\"><div class=\"clinical_share \"><div class=\"clinical_share news-bind\">\n    \t<div class=\"page-myBinder share-binder share-item\" data-tag=\"news-meeting\" data-id=\"98511\"><img src=\"https:\/\/www.onempsvoice.com\/application\/themes\/onevoice-child\/images\/bookmark.png\" style=\"width:20px;\">  Bookmark<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><div class=\"social-share-box\">\n                                                        <div class=\"social-share\"> <span class=\"icon-share\"><\/span> Share\n                                                        <div class=\"social-share-hov social-share-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"share-discuss share-item share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a href=\"\" class=\"page-discuss-courage\"><span class=\"icon-talk icon-left\"><\/span> Discuss in Social Wall<\/a>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"post-facebook courage-social-cont share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a  href=\"javascript:void(0)\" title=\"share on Facebook\" data-detailurl=\"https:\/\/www.onempsvoice.com\/rcuratenew\/open-label-phase-1-2-study-vestronidase-alfa-mucopolysaccharidosis-vii\/\" class=\"btnfbShare\"><div  class=\"head_fb header_facebook_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-facebook\"><\/i> <\/div>Facebook<\/a> \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t  <div class=\"post-twitter courage-social-cont share-item-inner\">\n\t\t\t\t\t\t  <a data-href=\"https:\/\/twitter.com\/intent\/tweet?source=&text=:%20\" title=\"share on Twitter\" onclick=\"window.open('https:\/\/twitter.com\/intent\/tweet?text='+ encodeURIComponent('Open-Label Phase 1\/2 Study of Vestronidase Alfa for Mucopolysaccharidosis VII') + ' on @oneMPSvoice ' + encodeURIComponent('')); return false;\"><div  class=\"head_tweet header_twitter_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-twitter\"><\/i>  <\/div>Twitter<\/a>\n\t\t\t\t\t\t  <\/div><div class=\"share-team share-item share_with_rareteam share-item-inner\" post=\"98511\" ><span class=\"share-team-icon icon-left\"><\/span>Share to rareTeam<\/div>\n                                                        <\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><\/div><div class=\"trial_readmore\"><a class=\"button_grey\"  data-id=\"98511\" href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/open-label-phase-1-2-study-vestronidase-alfa-mucopolysaccharidosis-vii\/\"  >\nread more\n<\/a><\/div>\n\n\n<\/div><\/div><div class=\"show_more_main\"><div class=\"show_more_wrap\"><span data-array='[\"98494\",\"98496\",\"98498\",\"98499\",\"98500\",\"98511\",\"98513\",\"98514\",\"98516\",\"98484\",\"98485\",\"98488\",\"98490\",\"98492\",\"98453\",\"98454\",\"98455\",\"98456\",\"98458\",\"98470\",\"98471\",\"98472\",\"98473\",\"98474\",\"98477\",\"98478\",\"98444\",\"98446\",\"98448\",\"98449\",\"98451\",\"98452\",\"98435\",\"98436\",\"98437\",\"98438\",\"98439\",\"98440\",\"98403\",\"98405\",\"98406\",\"98408\",\"98412\",\"98416\",\"98417\",\"98419\",\"98420\",\"98422\",\"98423\",\"98426\",\"98427\",\"98428\",\"98429\",\"98430\",\"98432\",\"98434\",\"98401\",\"98402\",\"98368\",\"98369\",\"98371\",\"98372\",\"98375\",\"98376\",\"98382\",\"98383\",\"98386\",\"98387\",\"98388\",\"98391\",\"98393\",\"98394\",\"98395\",\"98396\",\"98400\",\"98364\",\"98365\",\"98366\",\"98347\",\"98348\",\"98349\",\"98350\",\"98351\",\"98352\",\"98353\",\"98355\",\"98357\",\"98360\",\"98361\",\"98363\",\"98329\",\"98330\",\"98331\",\"98332\",\"98334\",\"98335\",\"98336\",\"98337\",\"98338\",\"98340\",\"98341\",\"98342\",\"98343\",\"98345\",\"98346\",\"98324\",\"98325\",\"98326\",\"98327\",\"98328\"]' class=\"show_more curate-readmore\" data-slug=scientific-articles data-type=\"evidance-education\" data-id=98511 data-limit=6 data-count=104>Show more <\/span><\/div><\/div><\/div><div class=\"tab-pane\" id=\"patient-education\"><div class=\"curate-inner-subtitle\">25 resources available:<\/div><div class=\"evidence_info_content ev_ed\" >\n\t\t\t\t<a class=\"info_title collapsed\"><\/a>\n<div class=\"\n-evidence info_title\" data-id=\"101560\"><a href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/understanding-mucopolysaccharidosis-vii-sly-syndrome\/\"><h2>Understanding Mucopolysaccharidosis Type VII (MPS VII) or Sly Syndrome<\/h2><\/a>\n<span class=\"info_title_sub_text\">Ultragenyx Pharmaceutical Inc.<\/span><\/div>\n<div class=\"info_cont_hide\">MPS VII, also called Sly syndrome, is a rare, life-threatening lysosomal storage disorder, caused by mutations of the GUSB gene, which results in a\u00a0 deficiency of the \u03b2-glucuronidase enzyme. \u03b2-glucuronidase plays a key role in the breakdown of glycosaminoglycans (GAGs), previously called mucopolysaccharides. The inability to properly \u00a0break down GAGs causes them to build up in the lysosomes.Signs and symptoms of MPS VII generally become more...<div class=\"evidence-share-row\"><div class=\"clinical_share \"><div class=\"clinical_share news-bind\">\n    \t<div class=\"page-myBinder share-binder share-item\" data-tag=\"news-meeting\" data-id=\"101560\"><img src=\"https:\/\/www.onempsvoice.com\/application\/themes\/onevoice-child\/images\/bookmark.png\" style=\"width:20px;\">  Bookmark<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><div class=\"social-share-box\">\n                                                        <div class=\"social-share\"> <span class=\"icon-share\"><\/span> Share\n                                                        <div class=\"social-share-hov social-share-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"share-discuss share-item share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a href=\"\" class=\"page-discuss-courage\"><span class=\"icon-talk icon-left\"><\/span> Discuss in Social Wall<\/a>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"post-facebook courage-social-cont share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a  href=\"javascript:void(0)\" title=\"share on Facebook\" data-detailurl=\"https:\/\/www.onempsvoice.com\/rcuratenew\/understanding-mucopolysaccharidosis-vii-sly-syndrome\/\" class=\"btnfbShare\"><div  class=\"head_fb header_facebook_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-facebook\"><\/i> <\/div>Facebook<\/a> \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t  <div class=\"post-twitter courage-social-cont share-item-inner\">\n\t\t\t\t\t\t  <a data-href=\"https:\/\/twitter.com\/intent\/tweet?source=&text=:%20\" title=\"share on Twitter\" onclick=\"window.open('https:\/\/twitter.com\/intent\/tweet?text='+ encodeURIComponent('Understanding Mucopolysaccharidosis Type VII (MPS VII) or Sly Syndrome') + ' on @oneMPSvoice ' + encodeURIComponent('')); return false;\"><div  class=\"head_tweet header_twitter_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-twitter\"><\/i>  <\/div>Twitter<\/a>\n\t\t\t\t\t\t  <\/div><div class=\"share-team share-item share_with_rareteam share-item-inner\" post=\"101560\" ><span class=\"share-team-icon icon-left\"><\/span>Share to rareTeam<\/div>\n                                                        <\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><\/div><div class=\"trial_readmore\"><a class=\"button_grey\"  data-id=\"101560\" href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/understanding-mucopolysaccharidosis-vii-sly-syndrome\/\"  >\nread more\n<\/a><\/div>\n\n\n<\/div><\/div><div class=\"evidence_info_content ev_ed\" >\n\t\t\t\t<a class=\"info_title collapsed\"><\/a>\n<div class=\"\n-evidence info_title\" data-id=\"101555\"><a href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/understanding-mps-vi-maroteaux-lamy-syndrome\/\"><h2>A Guide to Understanding MPS VI (Maroteaux-Lamy Syndrome)<\/h2><\/a>\n<span class=\"info_title_sub_text\">National MPS Society<\/span><\/div>\n<div class=\"info_cont_hide\">Mucopolysaccharidosis VI (MPS VI) is a very rare genetic disorder characterized by a large head, distinctive \u201ccoarse\u201d features, and a large tongue. It is also known as Maroteaux-Lamy syndrome, named after two French physicians, Dr. Pierre Maroteaux and his mentor, Dr. Maurice Emil Joseph Lamy, who first described the condition in 1963. Other names for MPS VI include polydystrophic dwarfism and arylsulfatase B deficiency.How Is MPS VI Diagnose...<div class=\"evidence-share-row\"><div class=\"clinical_share \"><div class=\"clinical_share news-bind\">\n    \t<div class=\"page-myBinder share-binder share-item\" data-tag=\"news-meeting\" data-id=\"101555\"><img src=\"https:\/\/www.onempsvoice.com\/application\/themes\/onevoice-child\/images\/bookmark.png\" style=\"width:20px;\">  Bookmark<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><div class=\"social-share-box\">\n                                                        <div class=\"social-share\"> <span class=\"icon-share\"><\/span> Share\n                                                        <div class=\"social-share-hov social-share-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"share-discuss share-item share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a href=\"\" class=\"page-discuss-courage\"><span class=\"icon-talk icon-left\"><\/span> Discuss in Social Wall<\/a>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"post-facebook courage-social-cont share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a  href=\"javascript:void(0)\" title=\"share on Facebook\" data-detailurl=\"https:\/\/www.onempsvoice.com\/rcuratenew\/understanding-mps-vi-maroteaux-lamy-syndrome\/\" class=\"btnfbShare\"><div  class=\"head_fb header_facebook_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-facebook\"><\/i> <\/div>Facebook<\/a> \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t  <div class=\"post-twitter courage-social-cont share-item-inner\">\n\t\t\t\t\t\t  <a data-href=\"https:\/\/twitter.com\/intent\/tweet?source=&text=:%20\" title=\"share on Twitter\" onclick=\"window.open('https:\/\/twitter.com\/intent\/tweet?text='+ encodeURIComponent('A Guide to Understanding MPS VI (Maroteaux-Lamy Syndrome)') + ' on @oneMPSvoice ' + encodeURIComponent('')); return false;\"><div  class=\"head_tweet header_twitter_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-twitter\"><\/i>  <\/div>Twitter<\/a>\n\t\t\t\t\t\t  <\/div><div class=\"share-team share-item share_with_rareteam share-item-inner\" post=\"101555\" ><span class=\"share-team-icon icon-left\"><\/span>Share to rareTeam<\/div>\n                                                        <\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><\/div><div class=\"trial_readmore\"><a class=\"button_grey\"  data-id=\"101555\" href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/understanding-mps-vi-maroteaux-lamy-syndrome\/\"  >\nread more\n<\/a><\/div>\n\n\n<\/div><\/div><div class=\"evidence_info_content ev_ed\" >\n\t\t\t\t<a class=\"info_title collapsed\"><\/a>\n<div class=\"\n-evidence info_title\" data-id=\"101551\"><a href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/understanding-mps-iv-morquio-syndrome\/\"><h2>A Guide to Understanding MPS IV (Morquio Syndrome)<\/h2><\/a>\n<span class=\"info_title_sub_text\">National MPS Society<\/span><\/div>\n<div class=\"info_cont_hide\">Mucopolysaccharidosis IV (MPS IV) is a rare genetic disorder characterized by short stature and severe bone disease. It is also known as Morquio syndrome, named after Dr. Morquio, a pediatrician in Montevideo, Uruguay. In 1929, Dr. Morquio described a family of four children affected by this condition. The same year, Dr. Brailsford from Birmingham, England, also described the same characteristics. Consequently, it is sometimes known as Morquio-Br...<div class=\"evidence-share-row\"><div class=\"clinical_share \"><div class=\"clinical_share news-bind\">\n    \t<div class=\"page-myBinder share-binder share-item\" data-tag=\"news-meeting\" data-id=\"101551\"><img src=\"https:\/\/www.onempsvoice.com\/application\/themes\/onevoice-child\/images\/bookmark.png\" style=\"width:20px;\">  Bookmark<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><div class=\"social-share-box\">\n                                                        <div class=\"social-share\"> <span class=\"icon-share\"><\/span> Share\n                                                        <div class=\"social-share-hov social-share-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"share-discuss share-item share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a href=\"\" class=\"page-discuss-courage\"><span class=\"icon-talk icon-left\"><\/span> Discuss in Social Wall<\/a>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"post-facebook courage-social-cont share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a  href=\"javascript:void(0)\" title=\"share on Facebook\" data-detailurl=\"https:\/\/www.onempsvoice.com\/rcuratenew\/understanding-mps-iv-morquio-syndrome\/\" class=\"btnfbShare\"><div  class=\"head_fb header_facebook_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-facebook\"><\/i> <\/div>Facebook<\/a> \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t  <div class=\"post-twitter courage-social-cont share-item-inner\">\n\t\t\t\t\t\t  <a data-href=\"https:\/\/twitter.com\/intent\/tweet?source=&text=:%20\" title=\"share on Twitter\" onclick=\"window.open('https:\/\/twitter.com\/intent\/tweet?text='+ encodeURIComponent('A Guide to Understanding MPS IV (Morquio Syndrome)') + ' on @oneMPSvoice ' + encodeURIComponent('')); return false;\"><div  class=\"head_tweet header_twitter_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-twitter\"><\/i>  <\/div>Twitter<\/a>\n\t\t\t\t\t\t  <\/div><div class=\"share-team share-item share_with_rareteam share-item-inner\" post=\"101551\" ><span class=\"share-team-icon icon-left\"><\/span>Share to rareTeam<\/div>\n                                                        <\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><\/div><div class=\"trial_readmore\"><a class=\"button_grey\"  data-id=\"101551\" href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/understanding-mps-iv-morquio-syndrome\/\"  >\nread more\n<\/a><\/div>\n\n\n<\/div><\/div><div class=\"evidence_info_content ev_ed\" >\n\t\t\t\t<a class=\"info_title collapsed\"><\/a>\n<div class=\"\n-evidence info_title\" data-id=\"101549\"><a href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/considering-stem-cell-transplantation-questions-ask-provider\/\"><h2>Are You Considering Stem Cell Transplantation? Questions to Ask Your Provider<\/h2><\/a>\n<span class=\"info_title_sub_text\">National MPS Society<\/span><\/div>\n<div class=\"info_cont_hide\">Stem cell transplantation can be an overwhelming prospect, especially for a family already facing the usually fresh diagnosis of MPS or related disease. Typically, the time line is short, and the amount of information that needs to be assimilated is daunting. A framework of questions for understanding transplant is presented here. The intent is to provide a list of questions you can bring to your health care provider, to help decide whether or no...<div class=\"evidence-share-row\"><div class=\"clinical_share \"><div class=\"clinical_share news-bind\">\n    \t<div class=\"page-myBinder share-binder share-item\" data-tag=\"news-meeting\" data-id=\"101549\"><img src=\"https:\/\/www.onempsvoice.com\/application\/themes\/onevoice-child\/images\/bookmark.png\" style=\"width:20px;\">  Bookmark<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><div class=\"social-share-box\">\n                                                        <div class=\"social-share\"> <span class=\"icon-share\"><\/span> Share\n                                                        <div class=\"social-share-hov social-share-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"share-discuss share-item share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a href=\"\" class=\"page-discuss-courage\"><span class=\"icon-talk icon-left\"><\/span> Discuss in Social Wall<\/a>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"post-facebook courage-social-cont share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a  href=\"javascript:void(0)\" title=\"share on Facebook\" data-detailurl=\"https:\/\/www.onempsvoice.com\/rcuratenew\/considering-stem-cell-transplantation-questions-ask-provider\/\" class=\"btnfbShare\"><div  class=\"head_fb header_facebook_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-facebook\"><\/i> <\/div>Facebook<\/a> \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t  <div class=\"post-twitter courage-social-cont share-item-inner\">\n\t\t\t\t\t\t  <a data-href=\"https:\/\/twitter.com\/intent\/tweet?source=&text=:%20\" title=\"share on Twitter\" onclick=\"window.open('https:\/\/twitter.com\/intent\/tweet?text='+ encodeURIComponent('Are You Considering Stem Cell Transplantation? Questions to Ask Your Provider') + ' on @oneMPSvoice ' + encodeURIComponent('')); return false;\"><div  class=\"head_tweet header_twitter_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-twitter\"><\/i>  <\/div>Twitter<\/a>\n\t\t\t\t\t\t  <\/div><div class=\"share-team share-item share_with_rareteam share-item-inner\" post=\"101549\" ><span class=\"share-team-icon icon-left\"><\/span>Share to rareTeam<\/div>\n                                                        <\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><\/div><div class=\"trial_readmore\"><a class=\"button_grey\"  data-id=\"101549\" href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/considering-stem-cell-transplantation-questions-ask-provider\/\"  >\nread more\n<\/a><\/div>\n\n\n<\/div><\/div><div class=\"evidence_info_content ev_ed\" >\n\t\t\t\t<a class=\"info_title collapsed\"><\/a>\n<div class=\"\n-evidence info_title\" data-id=\"101547\"><a href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/understanding-mps-i-hurler-scheie-syndromes\/\"><h2>A Guide to Understanding MPS I (Hurler, Hurler-Scheie, and Scheie Syndromes)<\/h2><\/a>\n<span class=\"info_title_sub_text\">National MPS Society<\/span><\/div>\n<div class=\"info_cont_hide\">What Is MPS I? Mucopolysaccharidosis I (MPS I) is a rare genetic disorder that affects the entire body. It is also known as Hurler syndrome, named after Dr. Gertrude Hurler, a general practitioner, who in 1919 first described a boy and girl with severe symptoms of the condition. In 1962, Dr. Harold Scheie, an ophthalmologist, described individuals who displayed attenuated (less severe) symptoms and primarily were affected by corneal clouding. Thi...<div class=\"evidence-share-row\"><div class=\"clinical_share \"><div class=\"clinical_share news-bind\">\n    \t<div class=\"page-myBinder share-binder share-item\" data-tag=\"news-meeting\" data-id=\"101547\"><img src=\"https:\/\/www.onempsvoice.com\/application\/themes\/onevoice-child\/images\/bookmark.png\" style=\"width:20px;\">  Bookmark<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><div class=\"social-share-box\">\n                                                        <div class=\"social-share\"> <span class=\"icon-share\"><\/span> Share\n                                                        <div class=\"social-share-hov social-share-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"share-discuss share-item share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a href=\"\" class=\"page-discuss-courage\"><span class=\"icon-talk icon-left\"><\/span> Discuss in Social Wall<\/a>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"post-facebook courage-social-cont share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a  href=\"javascript:void(0)\" title=\"share on Facebook\" data-detailurl=\"https:\/\/www.onempsvoice.com\/rcuratenew\/understanding-mps-i-hurler-scheie-syndromes\/\" class=\"btnfbShare\"><div  class=\"head_fb header_facebook_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-facebook\"><\/i> <\/div>Facebook<\/a> \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t  <div class=\"post-twitter courage-social-cont share-item-inner\">\n\t\t\t\t\t\t  <a data-href=\"https:\/\/twitter.com\/intent\/tweet?source=&text=:%20\" title=\"share on Twitter\" onclick=\"window.open('https:\/\/twitter.com\/intent\/tweet?text='+ encodeURIComponent('A Guide to Understanding MPS I (Hurler, Hurler-Scheie, and Scheie Syndromes)') + ' on @oneMPSvoice ' + encodeURIComponent('')); return false;\"><div  class=\"head_tweet header_twitter_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-twitter\"><\/i>  <\/div>Twitter<\/a>\n\t\t\t\t\t\t  <\/div><div class=\"share-team share-item share_with_rareteam share-item-inner\" post=\"101547\" ><span class=\"share-team-icon icon-left\"><\/span>Share to rareTeam<\/div>\n                                                        <\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><\/div><div class=\"trial_readmore\"><a class=\"button_grey\"  data-id=\"101547\" href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/understanding-mps-i-hurler-scheie-syndromes\/\"  >\nread more\n<\/a><\/div>\n\n\n<\/div><\/div><div class=\"evidence_info_content ev_ed\" >\n\t\t\t\t<a class=\"info_title collapsed\"><\/a>\n<div class=\"\n-evidence info_title\" data-id=\"101543\"><a href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/hunter-syndrome-after-diagnosis-what-next\/\"><h2>Hunter Syndrome &#8211; After Diagnosis: What Next?<\/h2><\/a>\n<span class=\"info_title_sub_text\">Takeda<\/span><\/div>\n<div class=\"info_cont_hide\">Talking About the Diagnosis It can be hard to explain the disease to others. You could say that your child has a long-term condition that, depending on the individual\u2019s needs, may require regular healthcare management. You may wish to tell only a few people who need to know, such as family, close friends and key people at school. When talking to your children about Hunter syndrome, you will need to judge how much information to give them withou...<div class=\"evidence-share-row\"><div class=\"clinical_share \"><div class=\"clinical_share news-bind\">\n    \t<div class=\"page-myBinder share-binder share-item\" data-tag=\"news-meeting\" data-id=\"101543\"><img src=\"https:\/\/www.onempsvoice.com\/application\/themes\/onevoice-child\/images\/bookmark.png\" style=\"width:20px;\">  Bookmark<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><div class=\"social-share-box\">\n                                                        <div class=\"social-share\"> <span class=\"icon-share\"><\/span> Share\n                                                        <div class=\"social-share-hov social-share-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"share-discuss share-item share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a href=\"\" class=\"page-discuss-courage\"><span class=\"icon-talk icon-left\"><\/span> Discuss in Social Wall<\/a>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"post-facebook courage-social-cont share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a  href=\"javascript:void(0)\" title=\"share on Facebook\" data-detailurl=\"https:\/\/www.onempsvoice.com\/rcuratenew\/hunter-syndrome-after-diagnosis-what-next\/\" class=\"btnfbShare\"><div  class=\"head_fb header_facebook_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-facebook\"><\/i> <\/div>Facebook<\/a> \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t  <div class=\"post-twitter courage-social-cont share-item-inner\">\n\t\t\t\t\t\t  <a data-href=\"https:\/\/twitter.com\/intent\/tweet?source=&text=:%20\" title=\"share on Twitter\" onclick=\"window.open('https:\/\/twitter.com\/intent\/tweet?text='+ encodeURIComponent('Hunter Syndrome &#8211; After Diagnosis: What Next?') + ' on @oneMPSvoice ' + encodeURIComponent('')); return false;\"><div  class=\"head_tweet header_twitter_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-twitter\"><\/i>  <\/div>Twitter<\/a>\n\t\t\t\t\t\t  <\/div><div class=\"share-team share-item share_with_rareteam share-item-inner\" post=\"101543\" ><span class=\"share-team-icon icon-left\"><\/span>Share to rareTeam<\/div>\n                                                        <\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><\/div><div class=\"trial_readmore\"><a class=\"button_grey\"  data-id=\"101543\" href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/hunter-syndrome-after-diagnosis-what-next\/\"  >\nread more\n<\/a><\/div>\n\n\n<\/div><\/div><div class=\"show_more_main\"><div class=\"show_more_wrap\"><span data-array='[\"101560\",\"101555\",\"101551\",\"101549\",\"101547\",\"101543\",\"101539\",\"101536\",\"101534\",\"101529\",\"101526\",\"101524\",\"101520\",\"101517\",\"101514\",\"101507\",\"101481\",\"101478\",\"101476\",\"101474\",\"101472\",\"101469\",\"101467\",\"101465\",\"101463\"]' class=\"show_more curate-readmore\" data-slug=patient-education data-type=\"evidance-education\" data-id=101543 data-limit=6 data-count=19>Show more <\/span><\/div><\/div><\/div><div class=\"tab-pane\" id=\"abstracts-and-posters\"><div class=\"curate-inner-subtitle\">10 resources available:<\/div><div class=\"evidence_info_content ev_ed\" >\n\t\t\t\t<a class=\"info_title collapsed\"><\/a>\n<div class=\"\n-evidence info_title\" data-id=\"98495\"><a href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/cervical-disorders-mucopolysaccharidosis-iva-morquio-disease\/\"><h2>Cervical Disorders in Mucopolysaccharidosis IVA-Morquio Disease<\/h2><\/a>\n<span class=\"info_title_sub_text\">World Neurosurgery<\/span><span class=\"info_title_sub_text\">Year: 2021<\/span><\/div>\n<div class=\"info_cont_hide\">Morquio disease or mucopolysaccharidosis type IVA (Online Mendelian Inheritance in Man No. 253000) is a rare autosomal recessive disease classified in the group of metabolism inborn errors. The glycosaminoglycans accumulate in chondrocytes, which disturbs bone growth and leads to skeletal manifestations, such as skeletal dysplasia and a short stature. In addition, the disproportionate growth of the trachea can lead to airway insufficiency.We repo...<div class=\"evidence-share-row\"><div class=\"clinical_share \"><div class=\"clinical_share news-bind\">\n    \t<div class=\"page-myBinder share-binder share-item\" data-tag=\"news-meeting\" data-id=\"98495\"><img src=\"https:\/\/www.onempsvoice.com\/application\/themes\/onevoice-child\/images\/bookmark.png\" style=\"width:20px;\">  Bookmark<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><div class=\"social-share-box\">\n                                                        <div class=\"social-share\"> <span class=\"icon-share\"><\/span> Share\n                                                        <div class=\"social-share-hov social-share-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"share-discuss share-item share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a href=\"\" class=\"page-discuss-courage\"><span class=\"icon-talk icon-left\"><\/span> Discuss in Social Wall<\/a>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"post-facebook courage-social-cont share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a  href=\"javascript:void(0)\" title=\"share on Facebook\" data-detailurl=\"https:\/\/www.onempsvoice.com\/rcuratenew\/cervical-disorders-mucopolysaccharidosis-iva-morquio-disease\/\" class=\"btnfbShare\"><div  class=\"head_fb header_facebook_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-facebook\"><\/i> <\/div>Facebook<\/a> \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t  <div class=\"post-twitter courage-social-cont share-item-inner\">\n\t\t\t\t\t\t  <a data-href=\"https:\/\/twitter.com\/intent\/tweet?source=&text=:%20\" title=\"share on Twitter\" onclick=\"window.open('https:\/\/twitter.com\/intent\/tweet?text='+ encodeURIComponent('Cervical Disorders in Mucopolysaccharidosis IVA-Morquio Disease') + ' on @oneMPSvoice ' + encodeURIComponent('')); return false;\"><div  class=\"head_tweet header_twitter_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-twitter\"><\/i>  <\/div>Twitter<\/a>\n\t\t\t\t\t\t  <\/div><div class=\"share-team share-item share_with_rareteam share-item-inner\" post=\"98495\" ><span class=\"share-team-icon icon-left\"><\/span>Share to rareTeam<\/div>\n                                                        <\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><\/div><div class=\"trial_readmore\"><a class=\"button_grey\"  data-id=\"98495\" href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/cervical-disorders-mucopolysaccharidosis-iva-morquio-disease\/\"  >\nread more\n<\/a><\/div>\n\n\n<\/div><\/div><div class=\"evidence_info_content ev_ed\" >\n\t\t\t\t<a class=\"info_title collapsed\"><\/a>\n<div class=\"\n-evidence info_title\" data-id=\"98504\"><a href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/genome-editing-mucopolysaccharidoses-mucolipidoses\/\"><h2>Genome Editing in Mucopolysaccharidoses and Mucolipidoses<\/h2><\/a>\n<span class=\"info_title_sub_text\">Progress in Molecular Biology and Translational Science<\/span><span class=\"info_title_sub_text\">Year: 2021<\/span><\/div>\n<div class=\"info_cont_hide\">Mucopolysaccharidoses (MPS) and mucolipidoses (ML) are disorders that alter lysosome function. While MPS are caused by mutation in enzymes that degrade glycosaminoglycans, the ML are disorders characterized by reduced function in the phosphotransferase enzyme. Multiple clinical features are associated with these diseases and the exact mechanisms that could explain such different clinical manifestations in patients are still unknown. Furthermore,...<div class=\"evidence-share-row\"><div class=\"clinical_share \"><div class=\"clinical_share news-bind\">\n    \t<div class=\"page-myBinder share-binder share-item\" data-tag=\"news-meeting\" data-id=\"98504\"><img src=\"https:\/\/www.onempsvoice.com\/application\/themes\/onevoice-child\/images\/bookmark.png\" style=\"width:20px;\">  Bookmark<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><div class=\"social-share-box\">\n                                                        <div class=\"social-share\"> <span class=\"icon-share\"><\/span> Share\n                                                        <div class=\"social-share-hov social-share-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"share-discuss share-item share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a href=\"\" class=\"page-discuss-courage\"><span class=\"icon-talk icon-left\"><\/span> Discuss in Social Wall<\/a>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"post-facebook courage-social-cont share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a  href=\"javascript:void(0)\" title=\"share on Facebook\" data-detailurl=\"https:\/\/www.onempsvoice.com\/rcuratenew\/genome-editing-mucopolysaccharidoses-mucolipidoses\/\" class=\"btnfbShare\"><div  class=\"head_fb header_facebook_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-facebook\"><\/i> <\/div>Facebook<\/a> \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t  <div class=\"post-twitter courage-social-cont share-item-inner\">\n\t\t\t\t\t\t  <a data-href=\"https:\/\/twitter.com\/intent\/tweet?source=&text=:%20\" title=\"share on Twitter\" onclick=\"window.open('https:\/\/twitter.com\/intent\/tweet?text='+ encodeURIComponent('Genome Editing in Mucopolysaccharidoses and Mucolipidoses') + ' on @oneMPSvoice ' + encodeURIComponent('')); return false;\"><div  class=\"head_tweet header_twitter_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-twitter\"><\/i>  <\/div>Twitter<\/a>\n\t\t\t\t\t\t  <\/div><div class=\"share-team share-item share_with_rareteam share-item-inner\" post=\"98504\" ><span class=\"share-team-icon icon-left\"><\/span>Share to rareTeam<\/div>\n                                                        <\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><\/div><div class=\"trial_readmore\"><a class=\"button_grey\"  data-id=\"98504\" href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/genome-editing-mucopolysaccharidoses-mucolipidoses\/\"  >\nread more\n<\/a><\/div>\n\n\n<\/div><\/div><div class=\"evidence_info_content ev_ed\" >\n\t\t\t\t<a class=\"info_title collapsed\"><\/a>\n<div class=\"\n-evidence info_title\" data-id=\"98510\"><a href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/mpsbase-comprehensive-repository-differentially-expressed-genes-mucopolysaccharidoses\/\"><h2>MPSBase: Comprehensive Repository of Differentially Expressed Genes for Mucopolysaccharidoses<\/h2><\/a>\n<span class=\"info_title_sub_text\">Molecular genetics and metabolism<\/span><span class=\"info_title_sub_text\">Year: 2021<\/span><\/div>\n<div class=\"info_cont_hide\">Mucopolysaccharidoses (MPS) are lysosomal storage diseases (LSDs) caused by the deficiency of enzymes essential for the metabolism of extracellular matrix components called glycosaminoglycans (GAGs). To understand the physiopathology and alterations due to the lysosomal accumulation resulting from enzymatic deficiencies and their secondary outcomes can improve the diagnosis and treatment of rare genetic diseases. This work presents a database for...<div class=\"evidence-share-row\"><div class=\"clinical_share \"><div class=\"clinical_share news-bind\">\n    \t<div class=\"page-myBinder share-binder share-item\" data-tag=\"news-meeting\" data-id=\"98510\"><img src=\"https:\/\/www.onempsvoice.com\/application\/themes\/onevoice-child\/images\/bookmark.png\" style=\"width:20px;\">  Bookmark<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><div class=\"social-share-box\">\n                                                        <div class=\"social-share\"> <span class=\"icon-share\"><\/span> Share\n                                                        <div class=\"social-share-hov social-share-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"share-discuss share-item share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a href=\"\" class=\"page-discuss-courage\"><span class=\"icon-talk icon-left\"><\/span> Discuss in Social Wall<\/a>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"post-facebook courage-social-cont share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a  href=\"javascript:void(0)\" title=\"share on Facebook\" data-detailurl=\"https:\/\/www.onempsvoice.com\/rcuratenew\/mpsbase-comprehensive-repository-differentially-expressed-genes-mucopolysaccharidoses\/\" class=\"btnfbShare\"><div  class=\"head_fb header_facebook_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-facebook\"><\/i> <\/div>Facebook<\/a> \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t  <div class=\"post-twitter courage-social-cont share-item-inner\">\n\t\t\t\t\t\t  <a data-href=\"https:\/\/twitter.com\/intent\/tweet?source=&text=:%20\" title=\"share on Twitter\" onclick=\"window.open('https:\/\/twitter.com\/intent\/tweet?text='+ encodeURIComponent('MPSBase: Comprehensive Repository of Differentially Expressed Genes for Mucopolysaccharidoses') + ' on @oneMPSvoice ' + encodeURIComponent('')); return false;\"><div  class=\"head_tweet header_twitter_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-twitter\"><\/i>  <\/div>Twitter<\/a>\n\t\t\t\t\t\t  <\/div><div class=\"share-team share-item share_with_rareteam share-item-inner\" post=\"98510\" ><span class=\"share-team-icon icon-left\"><\/span>Share to rareTeam<\/div>\n                                                        <\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><\/div><div class=\"trial_readmore\"><a class=\"button_grey\"  data-id=\"98510\" href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/mpsbase-comprehensive-repository-differentially-expressed-genes-mucopolysaccharidoses\/\"  >\nread more\n<\/a><\/div>\n\n\n<\/div><\/div><div class=\"evidence_info_content ev_ed\" >\n\t\t\t\t<a class=\"info_title collapsed\"><\/a>\n<div class=\"\n-evidence info_title\" data-id=\"98512\"><a href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/real-world-data-assessment-safety-home-based-hospital-outpatient-based-laronidase-enzyme-replacement-therapy-mucopolysaccharidosis-i\/\"><h2>Real-World Data Assessment of Safety of Home-Based and Hospital\/Outpatient-Based laronidase Enzyme Replacement Therapy for Mucopolysaccharidosis I<\/h2><\/a>\n<span class=\"info_title_sub_text\">Molecular genetics and metabolism reports<\/span><span class=\"info_title_sub_text\">Year: 2021<\/span><\/div>\n<div class=\"info_cont_hide\">Enzyme replacement therapy (ERT) is available for several lysosomal storage disorders to address the underlying lysosomal enzyme deficiencies that lead to the accumulation of undegraded substrates in multiple organ systems. A typical ERT regimen is a 2\u20136 h weekly intravenous infusion. ERT administered in a home setting has been reported by patients and caregivers to be both less stressful and more convenient than hospital or clinic-based admini...<div class=\"evidence-share-row\"><div class=\"clinical_share \"><div class=\"clinical_share news-bind\">\n    \t<div class=\"page-myBinder share-binder share-item\" data-tag=\"news-meeting\" data-id=\"98512\"><img src=\"https:\/\/www.onempsvoice.com\/application\/themes\/onevoice-child\/images\/bookmark.png\" style=\"width:20px;\">  Bookmark<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><div class=\"social-share-box\">\n                                                        <div class=\"social-share\"> <span class=\"icon-share\"><\/span> Share\n                                                        <div class=\"social-share-hov social-share-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"share-discuss share-item share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a href=\"\" class=\"page-discuss-courage\"><span class=\"icon-talk icon-left\"><\/span> Discuss in Social Wall<\/a>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"post-facebook courage-social-cont share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a  href=\"javascript:void(0)\" title=\"share on Facebook\" data-detailurl=\"https:\/\/www.onempsvoice.com\/rcuratenew\/real-world-data-assessment-safety-home-based-hospital-outpatient-based-laronidase-enzyme-replacement-therapy-mucopolysaccharidosis-i\/\" class=\"btnfbShare\"><div  class=\"head_fb header_facebook_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-facebook\"><\/i> <\/div>Facebook<\/a> \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t  <div class=\"post-twitter courage-social-cont share-item-inner\">\n\t\t\t\t\t\t  <a data-href=\"https:\/\/twitter.com\/intent\/tweet?source=&text=:%20\" title=\"share on Twitter\" onclick=\"window.open('https:\/\/twitter.com\/intent\/tweet?text='+ encodeURIComponent('Real-World Data Assessment of Safety of Home-Based and Hospital\/Outpatient-Based laronidase Enzyme Replacement Therapy for Mucopolysaccharidosis I') + ' on @oneMPSvoice ' + encodeURIComponent('')); return false;\"><div  class=\"head_tweet header_twitter_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-twitter\"><\/i>  <\/div>Twitter<\/a>\n\t\t\t\t\t\t  <\/div><div class=\"share-team share-item share_with_rareteam share-item-inner\" post=\"98512\" ><span class=\"share-team-icon icon-left\"><\/span>Share to rareTeam<\/div>\n                                                        <\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><\/div><div class=\"trial_readmore\"><a class=\"button_grey\"  data-id=\"98512\" href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/real-world-data-assessment-safety-home-based-hospital-outpatient-based-laronidase-enzyme-replacement-therapy-mucopolysaccharidosis-i\/\"  >\nread more\n<\/a><\/div>\n\n\n<\/div><\/div><div class=\"evidence_info_content ev_ed\" >\n\t\t\t\t<a class=\"info_title collapsed\"><\/a>\n<div class=\"\n-evidence info_title\" data-id=\"98518\"><a href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/ultrasound-findings-finger-wrist-knee-joints-mucopolysaccharidosis-type-i\/\"><h2>Ultrasound Findings of Finger, Wrist and Knee Joints in Mucopolysaccharidosis Type I<\/h2><\/a>\n<span class=\"info_title_sub_text\">Molecular genetics and metabolism<\/span><span class=\"info_title_sub_text\">Year: 2021<\/span><\/div>\n<div class=\"info_cont_hide\">Musculoskeletal findings in MPS can progress after enzyme replacement. Our aim was to examine synovial recesses, tendons, retinacula and pulleys using ultrasonography for structural and inflammatory changes.<div class=\"evidence-share-row\"><div class=\"clinical_share \"><div class=\"clinical_share news-bind\">\n    \t<div class=\"page-myBinder share-binder share-item\" data-tag=\"news-meeting\" data-id=\"98518\"><img src=\"https:\/\/www.onempsvoice.com\/application\/themes\/onevoice-child\/images\/bookmark.png\" style=\"width:20px;\">  Bookmark<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><div class=\"social-share-box\">\n                                                        <div class=\"social-share\"> <span class=\"icon-share\"><\/span> Share\n                                                        <div class=\"social-share-hov social-share-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"share-discuss share-item share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a href=\"\" class=\"page-discuss-courage\"><span class=\"icon-talk icon-left\"><\/span> Discuss in Social Wall<\/a>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"post-facebook courage-social-cont share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a  href=\"javascript:void(0)\" title=\"share on Facebook\" data-detailurl=\"https:\/\/www.onempsvoice.com\/rcuratenew\/ultrasound-findings-finger-wrist-knee-joints-mucopolysaccharidosis-type-i\/\" class=\"btnfbShare\"><div  class=\"head_fb header_facebook_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-facebook\"><\/i> <\/div>Facebook<\/a> \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t  <div class=\"post-twitter courage-social-cont share-item-inner\">\n\t\t\t\t\t\t  <a data-href=\"https:\/\/twitter.com\/intent\/tweet?source=&text=:%20\" title=\"share on Twitter\" onclick=\"window.open('https:\/\/twitter.com\/intent\/tweet?text='+ encodeURIComponent('Ultrasound Findings of Finger, Wrist and Knee Joints in Mucopolysaccharidosis Type I') + ' on @oneMPSvoice ' + encodeURIComponent('')); return false;\"><div  class=\"head_tweet header_twitter_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-twitter\"><\/i>  <\/div>Twitter<\/a>\n\t\t\t\t\t\t  <\/div><div class=\"share-team share-item share_with_rareteam share-item-inner\" post=\"98518\" ><span class=\"share-team-icon icon-left\"><\/span>Share to rareTeam<\/div>\n                                                        <\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><\/div><div class=\"trial_readmore\"><a class=\"button_grey\"  data-id=\"98518\" href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/ultrasound-findings-finger-wrist-knee-joints-mucopolysaccharidosis-type-i\/\"  >\nread more\n<\/a><\/div>\n\n\n<\/div><\/div><div class=\"evidence_info_content ev_ed\" >\n\t\t\t\t<a class=\"info_title collapsed\"><\/a>\n<div class=\"\n-evidence info_title\" data-id=\"98483\"><a href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/corneal-cloudiness-presenting-feature-mucopolysaccharidosis-type-i\/\"><h2>Corneal Cloudiness: A Presenting Feature of Mucopolysaccharidosis Type I<\/h2><\/a>\n<span class=\"info_title_sub_text\">Indian journal of pediatrics<\/span><span class=\"info_title_sub_text\">Year: 2021<\/span><\/div>\n<div class=\"info_cont_hide\">A 10-y-old \u2018short-statured\u2019 male presented to us with decreased vision in both the eyes since birth. Ocular examination revealed cloudy corneas in both the eyes (Fig. 1a, b). Family history was unremarkable. Suspecting it to be mucopolysaccharidosis, the child was referred to a pediatrician. Systemic examination revealed global developmental delay, short stature, abdominal distension, umbilical hernia, and hepatosplenomegaly with dysmorphic f...<div class=\"evidence-share-row\"><div class=\"clinical_share \"><div class=\"clinical_share news-bind\">\n    \t<div class=\"page-myBinder share-binder share-item\" data-tag=\"news-meeting\" data-id=\"98483\"><img src=\"https:\/\/www.onempsvoice.com\/application\/themes\/onevoice-child\/images\/bookmark.png\" style=\"width:20px;\">  Bookmark<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><div class=\"social-share-box\">\n                                                        <div class=\"social-share\"> <span class=\"icon-share\"><\/span> Share\n                                                        <div class=\"social-share-hov social-share-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"share-discuss share-item share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a href=\"\" class=\"page-discuss-courage\"><span class=\"icon-talk icon-left\"><\/span> Discuss in Social Wall<\/a>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"post-facebook courage-social-cont share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a  href=\"javascript:void(0)\" title=\"share on Facebook\" data-detailurl=\"https:\/\/www.onempsvoice.com\/rcuratenew\/corneal-cloudiness-presenting-feature-mucopolysaccharidosis-type-i\/\" class=\"btnfbShare\"><div  class=\"head_fb header_facebook_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-facebook\"><\/i> <\/div>Facebook<\/a> \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t  <div class=\"post-twitter courage-social-cont share-item-inner\">\n\t\t\t\t\t\t  <a data-href=\"https:\/\/twitter.com\/intent\/tweet?source=&text=:%20\" title=\"share on Twitter\" onclick=\"window.open('https:\/\/twitter.com\/intent\/tweet?text='+ encodeURIComponent('Corneal Cloudiness: A Presenting Feature of Mucopolysaccharidosis Type I') + ' on @oneMPSvoice ' + encodeURIComponent('')); return false;\"><div  class=\"head_tweet header_twitter_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-twitter\"><\/i>  <\/div>Twitter<\/a>\n\t\t\t\t\t\t  <\/div><div class=\"share-team share-item share_with_rareteam share-item-inner\" post=\"98483\" ><span class=\"share-team-icon icon-left\"><\/span>Share to rareTeam<\/div>\n                                                        <\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><\/div><div class=\"trial_readmore\"><a class=\"button_grey\"  data-id=\"98483\" href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/corneal-cloudiness-presenting-feature-mucopolysaccharidosis-type-i\/\"  >\nread more\n<\/a><\/div>\n\n\n<\/div><\/div><div class=\"show_more_main\"><div class=\"show_more_wrap\"><span data-array='[\"98495\",\"98504\",\"98510\",\"98512\",\"98518\",\"98483\",\"98441\",\"98410\",\"98381\",\"98392\"]' class=\"show_more curate-readmore\" data-slug=abstracts-and-posters data-type=\"evidance-education\" data-id=98483 data-limit=6 data-count=4>Show more <\/span><\/div><\/div><\/div><div class=\"tab-pane\" id=\"reference-materials\"><div class=\"curate-inner-subtitle\">30 resources available:<\/div><div class=\"evidence_info_content ev_ed\" >\n\t\t\t\t<a class=\"info_title collapsed\"><\/a>\n<div class=\"\n-evidence info_title\" data-id=\"98520\"><a href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/demographic-clinical-ancestry-characterization-large-cluster-mucopolysaccharidosis-iv-a-brazilian-northeast-region\/\"><h2>Demographic, Clinical, and Ancestry Characterization of a Large Cluster of Mucopolysaccharidosis IV A in the Brazilian Northeast Region<\/h2><\/a>\n<span class=\"info_title_sub_text\">American journal of medical genetics. Part A<\/span><span class=\"info_title_sub_text\">Year: 2021<\/span><\/div>\n<div class=\"info_cont_hide\">Mucopolysaccharidosis (MPS) IVA is a rare autosomal recessive disease with a highly variable distribution worldwide. Discrepancies in the incidence of MPS IVA among populations of different ethnicities are mostly attributed to founder effects. Demographic and clinical data from 28 MPS IVA patients, followed at a single center, and ancestry (Y chromosome and mitochondrial markers) of a subsample of 17 patients, most with the p.Ser341Arg (c.1023C&#038;g...<div class=\"evidence-share-row\"><div class=\"clinical_share \"><div class=\"clinical_share news-bind\">\n    \t<div class=\"page-myBinder share-binder share-item\" data-tag=\"news-meeting\" data-id=\"98520\"><img src=\"https:\/\/www.onempsvoice.com\/application\/themes\/onevoice-child\/images\/bookmark.png\" style=\"width:20px;\">  Bookmark<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><div class=\"social-share-box\">\n                                                        <div class=\"social-share\"> <span class=\"icon-share\"><\/span> Share\n                                                        <div class=\"social-share-hov social-share-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"share-discuss share-item share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a href=\"\" class=\"page-discuss-courage\"><span class=\"icon-talk icon-left\"><\/span> Discuss in Social Wall<\/a>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"post-facebook courage-social-cont share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a  href=\"javascript:void(0)\" title=\"share on Facebook\" data-detailurl=\"https:\/\/www.onempsvoice.com\/rcuratenew\/demographic-clinical-ancestry-characterization-large-cluster-mucopolysaccharidosis-iv-a-brazilian-northeast-region\/\" class=\"btnfbShare\"><div  class=\"head_fb header_facebook_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-facebook\"><\/i> <\/div>Facebook<\/a> \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t  <div class=\"post-twitter courage-social-cont share-item-inner\">\n\t\t\t\t\t\t  <a data-href=\"https:\/\/twitter.com\/intent\/tweet?source=&text=:%20\" title=\"share on Twitter\" onclick=\"window.open('https:\/\/twitter.com\/intent\/tweet?text='+ encodeURIComponent('Demographic, Clinical, and Ancestry Characterization of a Large Cluster of Mucopolysaccharidosis IV A in the Brazilian Northeast Region') + ' on @oneMPSvoice ' + encodeURIComponent('')); return false;\"><div  class=\"head_tweet header_twitter_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-twitter\"><\/i>  <\/div>Twitter<\/a>\n\t\t\t\t\t\t  <\/div><div class=\"share-team share-item share_with_rareteam share-item-inner\" post=\"98520\" ><span class=\"share-team-icon icon-left\"><\/span>Share to rareTeam<\/div>\n                                                        <\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><\/div><div class=\"trial_readmore\"><a class=\"button_grey\"  data-id=\"98520\" href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/demographic-clinical-ancestry-characterization-large-cluster-mucopolysaccharidosis-iv-a-brazilian-northeast-region\/\"  >\nread more\n<\/a><\/div>\n\n\n<\/div><\/div><div class=\"evidence_info_content ev_ed\" >\n\t\t\t\t<a class=\"info_title collapsed\"><\/a>\n<div class=\"\n-evidence info_title\" data-id=\"98506\"><a href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/early-late-brain-resonance-findings-two-siblings-hunter-syndrome\/\"><h2>Early and Late Brain Resonance Findings of Two Siblings With Hunter Syndrome<\/h2><\/a>\n<span class=\"info_title_sub_text\">Revista de Neurologia<\/span><span class=\"info_title_sub_text\">Year: 2021<\/span><\/div>\n<div class=\"info_cont_hide\">Mucopolysaccharidosis type II (MPS II) is a lysosomal disease caused by deficiency of the enzyme iduronate-2-sulfatase (IDS), linked to the X chromosome, producing a wide spectrum of clinical manifestations.<div class=\"evidence-share-row\"><div class=\"clinical_share \"><div class=\"clinical_share news-bind\">\n    \t<div class=\"page-myBinder share-binder share-item\" data-tag=\"news-meeting\" data-id=\"98506\"><img src=\"https:\/\/www.onempsvoice.com\/application\/themes\/onevoice-child\/images\/bookmark.png\" style=\"width:20px;\">  Bookmark<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><div class=\"social-share-box\">\n                                                        <div class=\"social-share\"> <span class=\"icon-share\"><\/span> Share\n                                                        <div class=\"social-share-hov social-share-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"share-discuss share-item share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a href=\"\" class=\"page-discuss-courage\"><span class=\"icon-talk icon-left\"><\/span> Discuss in Social Wall<\/a>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"post-facebook courage-social-cont share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a  href=\"javascript:void(0)\" title=\"share on Facebook\" data-detailurl=\"https:\/\/www.onempsvoice.com\/rcuratenew\/early-late-brain-resonance-findings-two-siblings-hunter-syndrome\/\" class=\"btnfbShare\"><div  class=\"head_fb header_facebook_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-facebook\"><\/i> <\/div>Facebook<\/a> \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t  <div class=\"post-twitter courage-social-cont share-item-inner\">\n\t\t\t\t\t\t  <a data-href=\"https:\/\/twitter.com\/intent\/tweet?source=&text=:%20\" title=\"share on Twitter\" onclick=\"window.open('https:\/\/twitter.com\/intent\/tweet?text='+ encodeURIComponent('Early and Late Brain Resonance Findings of Two Siblings With Hunter Syndrome') + ' on @oneMPSvoice ' + encodeURIComponent('')); return false;\"><div  class=\"head_tweet header_twitter_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-twitter\"><\/i>  <\/div>Twitter<\/a>\n\t\t\t\t\t\t  <\/div><div class=\"share-team share-item share_with_rareteam share-item-inner\" post=\"98506\" ><span class=\"share-team-icon icon-left\"><\/span>Share to rareTeam<\/div>\n                                                        <\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><\/div><div class=\"trial_readmore\"><a class=\"button_grey\"  data-id=\"98506\" href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/early-late-brain-resonance-findings-two-siblings-hunter-syndrome\/\"  >\nread more\n<\/a><\/div>\n\n\n<\/div><\/div><div class=\"evidence_info_content ev_ed\" >\n\t\t\t\t<a class=\"info_title collapsed\"><\/a>\n<div class=\"\n-evidence info_title\" data-id=\"98505\"><a href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/surgical-management-aortic-root-dilatation-patient-suffering-hunter-syndrome\/\"><h2>Surgical Management of an Aortic Root Dilatation in a Patient Suffering From Hunter Syndrome<\/h2><\/a>\n<span class=\"info_title_sub_text\">Interactive Cardiovascular and Thoracic Surgery<\/span><span class=\"info_title_sub_text\">Year: 2021<\/span><\/div>\n<div class=\"info_cont_hide\">Hunter syndrome is a rare disease leading to glycosaminoglycan accumulation in tissues. Multiple organs are involved, but prognosis is mainly conditioned by cardiac and respiratory failures. Cardiac valvular impairment is quite common but aortic root dilatation is rarely described. This article covers a case of surgical root replacement due to aortic valve insufficiency and aortic root dilatation documented with magnetic resonance and computed to...<div class=\"evidence-share-row\"><div class=\"clinical_share \"><div class=\"clinical_share news-bind\">\n    \t<div class=\"page-myBinder share-binder share-item\" data-tag=\"news-meeting\" data-id=\"98505\"><img src=\"https:\/\/www.onempsvoice.com\/application\/themes\/onevoice-child\/images\/bookmark.png\" style=\"width:20px;\">  Bookmark<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><div class=\"social-share-box\">\n                                                        <div class=\"social-share\"> <span class=\"icon-share\"><\/span> Share\n                                                        <div class=\"social-share-hov social-share-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"share-discuss share-item share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a href=\"\" class=\"page-discuss-courage\"><span class=\"icon-talk icon-left\"><\/span> Discuss in Social Wall<\/a>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"post-facebook courage-social-cont share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a  href=\"javascript:void(0)\" title=\"share on Facebook\" data-detailurl=\"https:\/\/www.onempsvoice.com\/rcuratenew\/surgical-management-aortic-root-dilatation-patient-suffering-hunter-syndrome\/\" class=\"btnfbShare\"><div  class=\"head_fb header_facebook_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-facebook\"><\/i> <\/div>Facebook<\/a> \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t  <div class=\"post-twitter courage-social-cont share-item-inner\">\n\t\t\t\t\t\t  <a data-href=\"https:\/\/twitter.com\/intent\/tweet?source=&text=:%20\" title=\"share on Twitter\" onclick=\"window.open('https:\/\/twitter.com\/intent\/tweet?text='+ encodeURIComponent('Surgical Management of an Aortic Root Dilatation in a Patient Suffering From Hunter Syndrome') + ' on @oneMPSvoice ' + encodeURIComponent('')); return false;\"><div  class=\"head_tweet header_twitter_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-twitter\"><\/i>  <\/div>Twitter<\/a>\n\t\t\t\t\t\t  <\/div><div class=\"share-team share-item share_with_rareteam share-item-inner\" post=\"98505\" ><span class=\"share-team-icon icon-left\"><\/span>Share to rareTeam<\/div>\n                                                        <\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><\/div><div class=\"trial_readmore\"><a class=\"button_grey\"  data-id=\"98505\" href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/surgical-management-aortic-root-dilatation-patient-suffering-hunter-syndrome\/\"  >\nread more\n<\/a><\/div>\n\n\n<\/div><\/div><div class=\"evidence_info_content ev_ed\" >\n\t\t\t\t<a class=\"info_title collapsed\"><\/a>\n<div class=\"\n-evidence info_title\" data-id=\"98503\"><a href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/cerebral-revascularization-child-mucopolysaccharidosis-type-i\/\"><h2>Case Report: Cerebral Revascularization in a Child With Mucopolysaccharidosis Type I<\/h2><\/a>\n<span class=\"info_title_sub_text\">Frontiers in Pediatrics<\/span><span class=\"info_title_sub_text\">Year: 2021<\/span><\/div>\n<div class=\"info_cont_hide\">Mucopolysaccharidosis (MPS) type I is a rare lysosomal storage disorder caused by an accumulation of glycosaminoglycans (GAGs) resulting in multisystem disease. Neurological morbidity includes hydrocephalus, spinal cord compression, and cognitive decline. While many neurological symptoms have been described, stroke is not a widely-recognized manifestation of MPS I. Accordingly, patients with MPS I are not routinely evaluated for stroke, and there...<div class=\"evidence-share-row\"><div class=\"clinical_share \"><div class=\"clinical_share news-bind\">\n    \t<div class=\"page-myBinder share-binder share-item\" data-tag=\"news-meeting\" data-id=\"98503\"><img src=\"https:\/\/www.onempsvoice.com\/application\/themes\/onevoice-child\/images\/bookmark.png\" style=\"width:20px;\">  Bookmark<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><div class=\"social-share-box\">\n                                                        <div class=\"social-share\"> <span class=\"icon-share\"><\/span> Share\n                                                        <div class=\"social-share-hov social-share-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"share-discuss share-item share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a href=\"\" class=\"page-discuss-courage\"><span class=\"icon-talk icon-left\"><\/span> Discuss in Social Wall<\/a>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"post-facebook courage-social-cont share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a  href=\"javascript:void(0)\" title=\"share on Facebook\" data-detailurl=\"https:\/\/www.onempsvoice.com\/rcuratenew\/cerebral-revascularization-child-mucopolysaccharidosis-type-i\/\" class=\"btnfbShare\"><div  class=\"head_fb header_facebook_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-facebook\"><\/i> <\/div>Facebook<\/a> \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t  <div class=\"post-twitter courage-social-cont share-item-inner\">\n\t\t\t\t\t\t  <a data-href=\"https:\/\/twitter.com\/intent\/tweet?source=&text=:%20\" title=\"share on Twitter\" onclick=\"window.open('https:\/\/twitter.com\/intent\/tweet?text='+ encodeURIComponent('Case Report: Cerebral Revascularization in a Child With Mucopolysaccharidosis Type I') + ' on @oneMPSvoice ' + encodeURIComponent('')); return false;\"><div  class=\"head_tweet header_twitter_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-twitter\"><\/i>  <\/div>Twitter<\/a>\n\t\t\t\t\t\t  <\/div><div class=\"share-team share-item share_with_rareteam share-item-inner\" post=\"98503\" ><span class=\"share-team-icon icon-left\"><\/span>Share to rareTeam<\/div>\n                                                        <\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><\/div><div class=\"trial_readmore\"><a class=\"button_grey\"  data-id=\"98503\" href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/cerebral-revascularization-child-mucopolysaccharidosis-type-i\/\"  >\nread more\n<\/a><\/div>\n\n\n<\/div><\/div><div class=\"evidence_info_content ev_ed\" >\n\t\t\t\t<a class=\"info_title collapsed\"><\/a>\n<div class=\"\n-evidence info_title\" data-id=\"98501\"><a href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/mucopolysaccharidoses-type-i-gene-therapy\/\"><h2>Mucopolysaccharidoses Type I Gene Therapy<\/h2><\/a>\n<span class=\"info_title_sub_text\">Journal of inherited metabolic disease<\/span><span class=\"info_title_sub_text\">Year: 2021<\/span><\/div>\n<div class=\"info_cont_hide\">Mucopolysaccharidoses type I (MPS I) is an inherited metabolic disease characterized by a malfunction of the \u03b1-l-iduronidase (IDUA) enzyme leading to the storage of glycosaminoglycans in the lysosomes. This disease has longtime been studied as a therapeutic target for those studying gene therapy and many studies have been done using various vectors to deliver the IDUA gene for corrective treatment. Many vectors have difficulties with efficacy an...<div class=\"evidence-share-row\"><div class=\"clinical_share \"><div class=\"clinical_share news-bind\">\n    \t<div class=\"page-myBinder share-binder share-item\" data-tag=\"news-meeting\" data-id=\"98501\"><img src=\"https:\/\/www.onempsvoice.com\/application\/themes\/onevoice-child\/images\/bookmark.png\" style=\"width:20px;\">  Bookmark<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><div class=\"social-share-box\">\n                                                        <div class=\"social-share\"> <span class=\"icon-share\"><\/span> Share\n                                                        <div class=\"social-share-hov social-share-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"share-discuss share-item share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a href=\"\" class=\"page-discuss-courage\"><span class=\"icon-talk icon-left\"><\/span> Discuss in Social Wall<\/a>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"post-facebook courage-social-cont share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a  href=\"javascript:void(0)\" title=\"share on Facebook\" data-detailurl=\"https:\/\/www.onempsvoice.com\/rcuratenew\/mucopolysaccharidoses-type-i-gene-therapy\/\" class=\"btnfbShare\"><div  class=\"head_fb header_facebook_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-facebook\"><\/i> <\/div>Facebook<\/a> \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t  <div class=\"post-twitter courage-social-cont share-item-inner\">\n\t\t\t\t\t\t  <a data-href=\"https:\/\/twitter.com\/intent\/tweet?source=&text=:%20\" title=\"share on Twitter\" onclick=\"window.open('https:\/\/twitter.com\/intent\/tweet?text='+ encodeURIComponent('Mucopolysaccharidoses Type I Gene Therapy') + ' on @oneMPSvoice ' + encodeURIComponent('')); return false;\"><div  class=\"head_tweet header_twitter_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-twitter\"><\/i>  <\/div>Twitter<\/a>\n\t\t\t\t\t\t  <\/div><div class=\"share-team share-item share_with_rareteam share-item-inner\" post=\"98501\" ><span class=\"share-team-icon icon-left\"><\/span>Share to rareTeam<\/div>\n                                                        <\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><\/div><div class=\"trial_readmore\"><a class=\"button_grey\"  data-id=\"98501\" href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/mucopolysaccharidoses-type-i-gene-therapy\/\"  >\nread more\n<\/a><\/div>\n\n\n<\/div><\/div><div class=\"evidence_info_content ev_ed\" >\n\t\t\t\t<a class=\"info_title collapsed\"><\/a>\n<div class=\"\n-evidence info_title\" data-id=\"98497\"><a href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/awake-airway-endoscopy-mucopolysaccharidosis\/\"><h2>Awake Airway Endoscopy in Mucopolysaccharidosis: A Case Report<\/h2><\/a>\n<span class=\"info_title_sub_text\">Brazilian Journal of Anesthesiology (Elsevier)<\/span><span class=\"info_title_sub_text\">Year: 2021<\/span><\/div>\n<div class=\"info_cont_hide\">Mucopolysaccharidosis (MPS) are a group of rare genetic inherited diseases with a progressive course due to the accumulation of glycosaminoglycans resulting in anatomic abnormalities and organ dysfunction, including the respiratory, cardiovascular, skeletal, and neurological systems that can increase the risk of anesthesia complications. Clinical manifestations are variable, multisystemic, and include severe morphological changes. The anesthetic...<div class=\"evidence-share-row\"><div class=\"clinical_share \"><div class=\"clinical_share news-bind\">\n    \t<div class=\"page-myBinder share-binder share-item\" data-tag=\"news-meeting\" data-id=\"98497\"><img src=\"https:\/\/www.onempsvoice.com\/application\/themes\/onevoice-child\/images\/bookmark.png\" style=\"width:20px;\">  Bookmark<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><div class=\"social-share-box\">\n                                                        <div class=\"social-share\"> <span class=\"icon-share\"><\/span> Share\n                                                        <div class=\"social-share-hov social-share-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"share-discuss share-item share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a href=\"\" class=\"page-discuss-courage\"><span class=\"icon-talk icon-left\"><\/span> Discuss in Social Wall<\/a>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"post-facebook courage-social-cont share-item-inner\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a  href=\"javascript:void(0)\" title=\"share on Facebook\" data-detailurl=\"https:\/\/www.onempsvoice.com\/rcuratenew\/awake-airway-endoscopy-mucopolysaccharidosis\/\" class=\"btnfbShare\"><div  class=\"head_fb header_facebook_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-facebook\"><\/i> <\/div>Facebook<\/a> \n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t  <div class=\"post-twitter courage-social-cont share-item-inner\">\n\t\t\t\t\t\t  <a data-href=\"https:\/\/twitter.com\/intent\/tweet?source=&text=:%20\" title=\"share on Twitter\" onclick=\"window.open('https:\/\/twitter.com\/intent\/tweet?text='+ encodeURIComponent('Awake Airway Endoscopy in Mucopolysaccharidosis: A Case Report') + ' on @oneMPSvoice ' + encodeURIComponent('')); return false;\"><div  class=\"head_tweet header_twitter_share  icon-left\" style=\"cursor:pointer;\"><i class=\"fa fa-twitter\"><\/i>  <\/div>Twitter<\/a>\n\t\t\t\t\t\t  <\/div><div class=\"share-team share-item share_with_rareteam share-item-inner\" post=\"98497\" ><span class=\"share-team-icon icon-left\"><\/span>Share to rareTeam<\/div>\n                                                        <\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div><\/div><div class=\"trial_readmore\"><a class=\"button_grey\"  data-id=\"98497\" href=\"https:\/\/www.onempsvoice.com\/rcuratenew\/awake-airway-endoscopy-mucopolysaccharidosis\/\"  >\nread more\n<\/a><\/div>\n\n\n<\/div><\/div><div class=\"show_more_main\"><div class=\"show_more_wrap\"><span data-array='[\"98520\",\"98506\",\"98505\",\"98503\",\"98501\",\"98497\",\"98493\",\"98487\",\"98486\",\"98467\",\"98482\",\"98481\",\"98476\",\"98469\",\"98468\",\"98466\",\"98464\",\"98463\",\"98460\",\"98459\",\"98447\",\"98445\",\"98414\",\"98367\",\"98398\",\"98397\",\"98389\",\"98379\",\"98378\",\"98377\"]' class=\"show_more curate-readmore\" data-slug=reference-materials data-type=\"evidance-education\" data-id=98497 data-limit=6 data-count=24>Show more <\/span><\/div><\/div><\/div><\/div>","tab_count":{"scientific-articles":110,"patient-education":25,"abstracts-and-posters":10,"reference-materials":30},"act_tabid":"scientific-articles"}