Mucopolysaccharidosis Type Iiib
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CLINICAL_TRIALS research · 2025-09-02Clinical Trial Update: An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB [Phase 1, Phase 2]
An open-label Phase 1/2 study of JR-446 for the treatment of Mucopolysaccharidosis type IIIB is currently recruiting.
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CLINICAL_TRIALS trial_result · 2023-08-09Clinical Trial Update: Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE [Phase 4]
Clinical trial update for ICV AX 250 in MPS IIIB shows assessment of safety, tolerability, and efficacy in a Phase 4 study currently enrolli…
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CLINICAL_TRIALS trial_result · 2023-01-18Clinical Trial Update: A Treatment Extension Study of Mucopolysaccharidosis Type IIIB [Phase 2]
A Phase 2 treatment extension study for Mucopolysaccharidosis Type IIIB (MPS IIIB) is being conducted, with the status and detailed results …
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CLINICAL_TRIALS trial_result · 2020-08-06Clinical Trial Update: A Treatment Study of Mucopolysaccharidosis Type IIIB [Phase 1, Phase 2]
Phase 1/2 clinical trial in mucopolysaccharidosis type IIIB (MPS IIIB) completed.
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CLINICAL_TRIALS research · 2017-02-03Clinical Trial Update: A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB
A completed retrospective chart review describing deceased patients with Mucopolysaccharidosis Type IIIB (Sanfilippo syndrome).
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CLINICAL_TRIALS trial_result · 2016-06-27Clinical Trial Update: Evaluation of Blood Brain Barrier Integrity and Structural Abnormalities in MPS IIIB Patients Using Multimodal Magnetic Resonance Imaging
A completed clinical study used multimodal MRI to assess blood-brain barrier integrity and brain structure in patients with Mucopolysacchari…
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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…