Mucopolysaccharidosis Type Iii
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CLINICAL_TRIALS research · 2026-04-06Clinical Trial Update: Study of Cannabidiol in Sanfilippo Syndrome [Phase 2, Phase 3]
A cannabidiol study for Sanfilippo syndrome (Mucopolysaccharidosis type III) is recruiting participants for Phase 2 and Phase 3 at the Lundq…
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CLINICAL_TRIALS research · 2026-02-11Clinical Trial Update: MPS (RaDiCo Cohort) (RaDiCo-MPS)
An update on the RaDiCo-MPS clinical trial cohort sponsored by INSERM, recruiting patients with several mucopolysaccharidosis types I-IX and…
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CLINICAL_TRIALS research · 2026-02-09Clinical Trial Update: Longitudinal Study of Neurodegenerative Disorders
This clinical trial update describes a longitudinal study of several neurodegenerative and lysosomal storage disorders. The study is current…
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CLINICAL_TRIALS research · 2025-08-20Clinical Trial Update: An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambroxol Hydrochloride in Adult (≥ 18 Years of Age) Subjects With MPS III [Phase 2, Phase 3]
An open-label dose-escalation study (Phase 2/Phase 3) is evaluating the safety, tolerability, and pharmacologic properties of high-dose ambr…
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CLINICAL_TRIALS trial_result · 2024-10-22Clinical Trial Update: Open-label Study of Anakinra in MPS III [Phase 1, Phase 2]
An open-label Phase 1/2 trial evaluating Anakinra for Mucopolysaccharidosis type III has completed.
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CLINICAL_TRIALS trial_result · 2017-12-13Clinical Trial Update: Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III
A clinical trial examining parental coping with challenging behavior in children with mucopolysaccharidosis types I–III has completed.