Sanfilippo Syndrome Type
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH [Phase 2, Phase 3]
Ultragenyx is conducting a Phase 2/3 gene therapy trial (scAAV9.U1a.hSGSH) for Mucopolysaccharidosis Type IIIA (Sanfilippo syndrome type A);…
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CLINICAL_TRIALS research · 2025-12-05Clinical Trial Update: Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A) [Phase 1, Phase 2]
Update on a Phase 1/2 clinical trial of DNL126 in pediatric patients with Mucopolysaccharidosis Type IIIA (Sanfilippo syndrome type A); the …
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CLINICAL_TRIALS research · 2025-08-28Clinical Trial Update: Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA) [Phase 1]
Phase 1 study of GC1130A for Sanfilippo syndrome type A is currently recruiting, indicating early-stage investigation of the therapy.
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CLINICAL_TRIALS research · 2025-08-01Clinical Trial Update: Natural History Study of Participants With Sanfilippo Syndrome Type IIIC
This clinical trial update describes a natural history study for Sanfilippo syndrome type C that is currently recruiting.
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CLINICAL_TRIALS research · 2025-01-30Clinical Trial Update: A Natural History Study of Biomarkers and Clinical Outcomes in Mucopolysaccharidosis Type IIIA (MPS IIIA; Sanfilippo Syndrome)
This ClinicalTrials.gov update notes the completion of a natural history study evaluating biomarkers and clinical outcomes in Mucopolysaccha…
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