Neurometabolic: All Phases trials

Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease

2-hydroxypropyl-beta-cyclodextrin

Phase I

United States

Recruiting

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Clinical Study to Evaluate the Safety and Efficacy BMMNC in Cerebellar Ataxia

Intravenous transfer of of Bone Marrow derived Mono Nuclear Stem Cell (BMMNCs)

Phase I

India

Recruiting

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An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165 (Prism301)

BMN 165

Phase III

United States

Recruiting

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Intramuscular Infusion of Autologous Bone Marrow Stem Cells in Patients With Amyotrophic Lateral Sclerosis (TCIM/ELA)

MNC (Mononuclear cells)

Phase I

Spain

Recruiting

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