Sanfilippo Syndrome
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CLINICAL_TRIALS research · 2026-04-06Clinical Trial Update: Study of Cannabidiol in Sanfilippo Syndrome [Phase 2, Phase 3]
A cannabidiol study for Sanfilippo syndrome (Mucopolysaccharidosis type III) is recruiting participants for Phase 2 and Phase 3 at the Lundq…
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CLINICAL_TRIALS research · 2025-08-20Clinical Trial Update: An 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 III [Phase 2, Phase 3]
An open-label dose-escalation study (Phase 2/Phase 3) is evaluating the safety, tolerability, and pharmacologic properties of high-dose ambr…