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Condition: Mucopolysaccharidosis Iii Synced from ClinicalTrials.gov · July 29, 2026

Recruiting studies: Mucopolysaccharidosis Iii

Studies currently listed as recruiting on the public registry for this search term. Each page explains the study in plain language, with locations and the study team's contact details. Clinicians: generate a printable list for your patient →

An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB
Phase 1/Phase 2 · 3 location(s) · JCR Pharmaceuticals Co., Ltd. · NCT06488924Natural History Study of Participants With Sanfilippo Syndrome Type IIIC
Phase N/A · 2 location(s) · Phoenix Nest · NCT05825131Study of Cannabidiol in Sanfilippo Syndrome
Phase 2/Phase 3 · 1 location(s) · Lundquist Institute for Biomedical Innovation at Harbor-UCLA · NCT06333041MPS (RaDiCo Cohort) (RaDiCo-MPS)
Phase N/A · 23 location(s) · Institut National de la Santé Et de la Recherche Médicale, F · NCT06036693Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
Phase 2/Phase 3 · 5 location(s) · Ultragenyx Pharmaceutical Inc · NCT02716246Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Phase 1 · 5 location(s) · GC Biopharma Corp · NCT06567769An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambrox
Phase 2/Phase 3 · 1 location(s) · Ozlem Goker-Alpan · NCT06614894Longitudinal Study of Neurodegenerative Disorders
Phase N/A · 1 location(s) · University of Pittsburgh · NCT03333200
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This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.