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Trusted Resources: News & Meetings

Latest announcements and gatherings

9 resources available:

Ultragenyx Acquires Global Rights to AAV Gene Therapy ABO-102 for Sanfilippo Syndrome Type A (MPS IIIA) From Abeona Therapeutics

Denali Therapeutics Announces Continued Progress In DNL310 (ETV:IDS) Program For MPS II (Hunter Syndrome) Supporting Planned Initiation Of Phase 2/3 Clinical Trial

Denali Therapeutics Announces Continued Progress in DNL310 (ETV:IDS) Program for MPS II (Hunter Syndrome) Supporting Planned Initiation of Phase 2/3 Clinical Trial

Regenxbio Presents Positive Initial Data From Phase I/II Trial Of Rgx-111 For The Treatment Of Severe MPS I At 18th Annual World Symposium™ 2022

Regenxbio Presents Positive Initial Data From Phase I/II Trial Of Rgx-111 for the Treatment of Severe MPS I at 18th Annual World Symposium™ 2022

Experimental MPS 1 Therapy Shows Biomarker Activity, Improved Function

Experimental MPS 1 Therapy Shows Biomarker Activity, Improved Function

New Type A ERT Candidate On Track For Clinical Trial

New Type A ERT Candidate on Track for Clinical Trial

Sanfilippo Ideal For FDA’s Accelerated Approval Pathway, Foundation Says

Sanfilippo Ideal for FDA’s Accelerated Approval Pathway, Foundation Says

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8 resources available:

Full Letter From Takeda to the National MPS Society

Advocacy Groups Unveil ‘Roadmap’ For Sanfilippo Research, Care

Advocacy Groups Unveil ‘Roadmap’ for Sanfilippo Research, Care

Europe Grants Orphan Drug Status To JR-441, ERT For Sanfilippo A

Europe Grants Orphan Drug Status to JR-441, ERT for Sanfilippo A

Luna And Partners Work To Accelerate Therapies For Hunter Syndrome

Luna and Partners Work to Accelerate Therapies for Hunter Syndrome

EMA Grants Orphan Drug Designation to JR-171 for the Treatment of Mucopolysaccaridosis Type I (MPS I)

Gene Therapy For Sanfilippo: Where We Are Now

Gene Therapy for Sanfilippo: Where We Are Now

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5 resources available:

Hurler Syndrome Treatment Market Research Prophesied To Grow At A Faster Pace By 2026

Hurler Syndrome Treatment Market Research Prophesied to Grow at a Faster Pace By 2026

Mucopolysaccharidosis Market: Global Industry Analysis And Opportunity Assessment 2022-2029

Mucopolysaccharidosis Market: Global Industry Analysis and Opportunity Assessment 2022-2029

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) Market Research Report – Global Clinical Trials Review, H2, 2021 – ResearchAndMarkets.com

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) Market Research Report – Global Clinical Trials Review, H2, 2021 – ResearchAndMarkets.com

Update From Mucolipidosis Collaborative Network (MCRN) And Cure Mucolipidosis (Cure ML) On Mucolipidosis Type II

Update from Mucolipidosis Collaborative Network (MCRN) and Cure Mucolipidosis (Cure ML) on Mucolipidosis Type II

Takeda to Commercialize Next-Generation Hunter Syndrome Therapy Through Collaboration With JCR Pharmaceuticals

7 resources available:

A 22 Year Old Rotherham Man, No Longer Walk Up His Stairs

A 22 Year Old Rotherham Man, no Longer Walk Up His Stairs

York Hob Moor Oaks School Boy Battles Hunter Syndrome

York Hob Moor Oaks School Boy Battles Hunter Syndrome

Losing Stanley – My Little Boy Doesn’t Recognise Me Anymore

Losing Stanley – My Little Boy Doesn’t Recognise Me Anymore

How One Mother Turned Her Son’s Rare Disease Diagnosis Into An Opportunity To Help Others

How One Mother Turned Her Son’s Rare Disease Diagnosis Into an Opportunity to Help Others

Announcing Tracy Kirby, Director of Development

Stem Cell Transplant For Bridgnorth Boy With Rare Condition

Stem Cell Transplant for Bridgnorth Boy With Rare Condition

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2 resources available:

36th Annual Family Conference: Nashville, TN

36th Annual Family Conference: Nashville, TN

H4B Boston’s Living Rooms Of Rare

H4B Boston’s Living Rooms of Rare

4 resources available:

Fall Family Fundraising

Fall Family Fundraising

Believe In G Golf Tournament

Believe in G Golf Tournament

Oliver’s Tomorrow Night At Columbus Blue Jackets

Oliver’s Tomorrow Night at Columbus Blue Jackets

Raleigh Run for Rare