Showing 10 of 10 recruiting trials for “Mucopolysaccharidosis”
An 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
Enrolling by InvitationNCT06103487 ↗
Long Term Follow-Up for RGX-111
ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I
An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects
A 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)
👨⚕️ Jose Alcantara Rodriguez, PharmD, Denali Therapeutics Inc.📍 32 sites📅 Started Jul 2022View details ↗
Registry of Patients Diagnosed With Lysosomal Storage Diseases
👨⚕️ Tippi C MacKenzie, MD, University of California, San Francisco📍 1 site📅 Started May 2022View details ↗
A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II
RecruitingNCT05284006 ↗
Non-invasive Functional Assessment and Pathogenesis of Morquio A
Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA
RecruitingNCT03604835 ↗
Mucopolysaccharidosis VII Disease Monitoring Program
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