Mucopolysaccharidosis Type Iiia
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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 · 2025-08-26Clinical Trial Update: Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA [Phase 1, Phase 2]
Phase 1/2 study of JR-441 in patients with Mucopolysaccharidosis Type IIIA is currently active but not recruiting.
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CLINICAL_TRIALS research · 2025-03-30Clinical Trial Update: Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA [Phase 1, Phase 2]
Phase 1/2 clinical trial evaluating a gene therapy using modified autologous hematopoietic stem cells for Mucopolysaccharidosis Type IIIA; s…
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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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