Clinical trials for Sanfilippo Syndrome
2 recruiting or active trials synced from ClinicalTrials.gov.
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Clinical trial records are synced from ClinicalTrials.gov through automated extraction.
Report missing data- RECRUITINGPhase 2NCT06614894An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambroxol Hydrochloride in Adult (≥ 18 Years of Age) Subjects With MPS IIIOzlem Goker-Alpan · PI: Ozlem Goker-Alpan, MD
- RECRUITINGPHASE2, PHASE3NCT02716246Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSHUltragenyx Pharmaceutical Inc · PI: Medical Director