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 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 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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CLINICAL_TRIALS trial_result · 2023-07-25Clinical Trial Update: Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease [Phase 1, Phase 2]
A phase 1/2 clinical trial of ABO-102 for MPS IIIA (Sanfilippo syndrome) was terminated; results have been posted.
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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 · 2021-08-31Clinical Trial Update: Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA) [Phase 2, Phase 3]
A study of AAVrh10-h.SGSH gene therapy in MPS IIIA is in Phase 2/3 with status not provided.
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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 · 2020-04-20Clinical Trial Update: An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093. [Phase 2]
An extension study to assess safety and efficacy in pediatric MPS IIIA patients who participated in a prior study (Phase 2; terminated).
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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 · 2015-11-16Clinical Trial Update: Natural History Studies of Mucopolysaccharidosis III
The document reports the completion of natural history studies for Mucopolysaccharidosis Type IIIA and Type IIIB, sponsored by Nationwide Ch…
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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…