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 trial_result · 2026-04-24Clinical Trial Update: Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA [Phase 3]
Phase 3 follow-up study of AAV-mediated gene transfer (UX111, formerly ABO-102) for Mucopolysaccharidosis type IIIA is currently enrolling b…
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CLINICAL_TRIALS trial_result · 2026-01-07Clinical Trial Update: MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis [Phase 2]
Phase 2 trial update describing allogeneic hematopoietic cell transplantation for metabolic disorders and severe osteopetrosis, with the stu…
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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 trial_result · 2025-08-01Clinical Trial Update: A Natural History Study of Sanfilippo Syndrome Type D
This clinical trial update reports that the natural history study of Sanfilippo Syndrome Type D (NCT05648851) sponsored by Phoenix Nest has …
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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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CLINICAL_TRIALS trial_result · 2022-02-25Clinical Trial Update: A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients [Phase 1, Phase 2]
Clinical trial update for SOBI003 in pediatric Sanfilippo Syndrome Type A (MPS IIIA) reporting completion with results posted.
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CLINICAL_TRIALS trial_result · 2021-04-22Clinical Trial Update: A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients [Phase 1, Phase 2]
A clinical trial assessing safety and tolerability of SOBI003 in pediatric patients with Sanfilippo Syndrome Type A (MPS IIIA) has completed…
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CLINICAL_TRIALS trial_result · 2020-04-21Clinical Trial Update: Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA) [Phase 1, Phase 2]
Extension study of rhHNS in Sanfilippo Syndrome Type A (MPS IIIA) in Phase 1/2; trial terminated with results posted.
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CLINICAL_TRIALS trial_result · 2019-12-02Clinical Trial Update: Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome [Phase 1, Phase 2]
A phase 1/2 clinical trial investigating intracerebral gene therapy for Sanfilippo syndrome type B has completed.
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CLINICAL_TRIALS trial_result · 2017-06-20Clinical Trial Update: Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy [Phase 1, Phase 2]
This update reports long-term follow-up results from a Phase 1/2 trial of SAF-301 gene therapy administered intracerebrally in patients with…
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CLINICAL_TRIALS trial_result · 2016-03-30Clinical Trial Update: A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)
A completed clinical trial for Sanfilippo Syndrome Type A (MPS IIIA) with posted results.
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CLINICAL_TRIALS trial_result · 2014-05-06Clinical Trial Update: Intracerebral Gene Therapy for Sanfilippo Type A Syndrome [Phase 1, Phase 2]
The Phase 1/2 intracerebral gene therapy trial for Sanfilippo syndrome type A (Mucopolysaccharidosis type IIIA) sponsored by Lysogene has co…