Mucopolysaccharidosis

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At a Glance

Live from database

Regulatory Watchboard

10 events
Mar 2026Physical Activity Intervention for MPS

Brunel University

TrialNOT YET RECRUITING
Aug 2025Nutritional Assessment in Patient of Mucopolysaccharide "

Assiut University — NA

TrialNOT YET RECRUITING
Sep 2023An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007

Denali Therapeutics Inc. — PHASE2, PHASE3

TrialENROLLING BY INVITATION
Jul 2023Long Term Follow-Up for RGX-111

REGENXBIO Inc.

TrialENROLLING BY INVITATION
Apr 2023ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I

Immusoft of CA, Inc. — PHASE1

TrialRECRUITING
Feb 2023An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects

JCR Pharmaceuticals Co., Ltd. — PHASE3

TrialENROLLING BY INVITATION
Sep 2022A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ

GC Biopharma Corp — PHASE1

TrialACTIVE NOT RECRUITING
Jul 2022A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Denali Therapeutics Inc. — PHASE2, PHASE3

TrialRECRUITING
May 2022Registry of Patients Diagnosed With Lysosomal Storage Diseases

University of California, San Francisco — NA

TrialRECRUITING
Feb 2022A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)

JCR Pharmaceuticals Co., Ltd. — PHASE3

TrialACTIVE NOT RECRUITING

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

4 programs

FINANCIAL LANDSCAPE SUMMARY

4

Total programs

3

Open now

1

Copay cards

3

Travel grants

Copay Assistance1

MEPSEVII

Ultragenyx

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Travel Grants3

Accessia Health: MPS-1 - Public Insurance

Accessia Health

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Accessia Health: MPS-1 - Public Insurance: Waitlist

Accessia Health

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The Assistance Fund (TAF) Financial Assistance Program

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Clinical Trial Landscape

16 active trials
3Phase 3
2Phase 2
2Phase 1
1N/A
3Unknown
2PHASE1, PHASE2
3PHASE2, PHASE3
16Total recruiting
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Recent News & Research

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Specialist Network

Top 6 by expertise

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