Records containing: Mucopolysaccharidosis III
This is the wording recorded or indexed by the source. An agency index term is not necessarily an authorised indication. Co-listed drug names are not linked to this term as a treatment relationship.
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CLINICAL_TRIALS study record update · 2026-04-24Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA
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CLINICAL_TRIALS study record update · 2026-04-06Randomized, Double-Blind, Placebo-Controlled Cross-Over Study to Evaluate the Effects of Cannabidiol on Neurobehavioral and Function Outcomes in Sanfilippo Syndrome
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CLINICAL_TRIALS study record update · 2026-02-11Mucopolysaccharidosis Patients in France in the Era of Specific Therapeutics
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CLINICAL_TRIALS study record update · 2024-10-22Open-label Pilot Study of the Effects of Anakinra in Mucopolysaccharidosis (MPS) III
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CLINICAL_TRIALS study record update · 2023-07-25A Phase I/II Open Label, Single-dose, Gene Transfer Study of scAAV9.U1a.hSGSH (ABO-102) in Patients With Middle and Advanced Phases of MPS IIIA Disease
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