Clinical Trials

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Clinical Trial
  • Status: Recruiting
  • Phase: Phase 3
  • Intervention Type: Drug
  • Participants: 62
  • Start Date: June 29, 2020
A Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, Safety, and Tolerability of Venglustat in Late-onset GM2 Gangliosidosis (Tay-Sachs Disease and Sandhoff Disease) Together With a Separate Basket for Juvenile/Adolescent Late-onset GM2 Gangliosidosis and Ultra-rare Diseases Within the Same and Similar Glucosylceramide-based Sphingolipid Pathway
Clinical Trial
  • Status: Active, not recruiting
  • Participants: 1000
  • Start Date: August 20, 2018
Biomarker for Glycogen Storage Diseases - AN INTERNATIONAL, MULTICENTER, EPIDEMIOLOGICAL PROTOCOL
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Clinical Trial
  • Status: Active, not recruiting
  • Participants: 1000
  • Start Date: August 20, 2018
Biomarker for Mucolipidosis Disorder Type I, II, III, IV AN INTERNATIONAL, MULTICENTER, EPIDEMIOLOGICAL PROTOCOL
Clinical Trial
  • Status: Recruiting
  • Participants: 20000
  • Start Date: July 2010
Coordination of Rare Diseases at Sanford
Clinical Trial
  • Status: Recruiting
  • Participants: 200
  • Start Date: January 25, 2002
Investigation of Neurodegeneration in Glycosphingolipid Storage Disorders
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