Mucopolysaccharidosis type 1

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At a Glance

Live from database

Regulatory Watchboard

10 events
Sep 2025Cardiac Structure and Function in MPS

Children's Hospital of Orange County

TrialRECRUITING
Nov 2024An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB

JCR Pharmaceuticals Co., Ltd. — PHASE1, PHASE2

TrialRECRUITING
Jul 2024A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 in Patients With Mucopolysaccharidosis Type I

West China Hospital — PHASE1

TrialRECRUITING
Dec 2023Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)

Denali Therapeutics Inc. — PHASE1, PHASE2

TrialACTIVE NOT RECRUITING
Oct 2023Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA

JCR Pharmaceuticals Co., Ltd. — PHASE1, PHASE2

TrialACTIVE NOT RECRUITING
Jun 2023Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type II

University of Manchester — PHASE1, PHASE2

TrialACTIVE NOT RECRUITING
Nov 2022Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.

Masonic Cancer Center, University of Minnesota

TrialACTIVE NOT RECRUITING
Jul 2021PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

University of California, San Francisco — PHASE1

TrialRECRUITING
Jan 2020Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA

University of Manchester — PHASE1, PHASE2

TrialACTIVE NOT RECRUITING
Sep 2018CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)

REGENXBIO Inc. — PHASE2, PHASE3

TrialACTIVE NOT RECRUITING

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

Financial Assistance

FINANCIAL LANDSCAPE SUMMARY

0

Total programs

No financial assistance programs currently listed for Mucopolysaccharidosis type 1.
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Clinical Trial Landscape

9 active trials
1Phase 1
1Unknown
6PHASE1, PHASE2
1PHASE2, PHASE3
9Total recruiting
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Recent News & Research

No recent news articles indexed yet for Mucopolysaccharidosis type 1.
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Specialist Network

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