Trusted Resources: People & Places
Online groups, photo galleries and blogs
CHOP – Lysosomal Storage Disease (LSD) Center3401 Civic Center Blvd.
Philadelphia, Pennsylvania, United States
The Lysosomal Storage Disease Center at Children’s Hospital of Philadelphia (CHOP) provides comprehensive testing, treatment and management for children, adolescents and young adults with a diagnosis or suspected diagnosis of lysosomal storage diseases (LSD) like Mucopolysaccharidosis.
Treatment for LSDs includes enzyme replacement therapy and bone marrow transplants to maximize patient outcomes. At CHOP’s LSD Center, we work with families to coordinate services across a therapeutic team.
Why Choose Us?
The Lysosomal Storage Disease Center combines excellent clinical care with access to active clinical research and new drug trials for patients interested in participating. Our team has been involved in LSD diagnosis and management for more than 20 years and has been recognized as a center for excellence. Our multidisciplinary clinics are devoted to our patients and aim to improve patient outcomes and satisfaction.
Conditions We Treat:
• Mucopolysaccharidosis Type 1
• Mucopolysaccharidosis Type II
A Review of the Clinical Outcomes in Idursulfase-Treated and Untreated Filipino Patients With Mucopolysaccharidosis ...Mucopolysaccharidosis type II (MPS II; H...
New Type A ERT Candidate on Track for Clinical TrialJapanese company, JCR Pharmaceuticals, h...
Open-Label Phase 1/2 Study of Vestronidase Alfa for Mucopolysaccharidosis VIIVestronidase alfa is an enzyme replaceme...
Emily Wants to Show Other Caregivers the Importance of Taking Care of YourselfPeople often make admiring comments to c...
Expanding the Phenotype of Mucopolysaccharidosis Type II RetinopathyPurpose: To report novel retinal finding...
Experimental MPS 1 Therapy Shows Biomarker Activity, Improved FunctionREGENXBIO presented results from its ong...
Airway Management in Mucopolysaccharidosis: A Retrospective Case Series ReviewAirway management can be difficult in su...