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Clinical trial condition

Lysosomal Storage Diseases

Explore clinical trials studying Lysosomal Storage Diseases. Study availability and eligibility vary by location and protocol.

717 public trials 126 recruiting studies
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Lysosomal Storage Diseases trial results

Recruiting

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

NCT07123155

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +17 more
Locations
  • University of California - Irvine Medical Center Irvine, California, United States
  • University of Florida (UF) - Gainesville Gainesville, Florida, United States
  • Emory University Hospital Atlanta, Georgia, United States
  • Washington University in St. Louis St Louis, Missouri, United States
View all 28 locations
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Recruiting

Pompe Disease Registry Protocol

NCT00231400

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • University of Alabama at Birmingham- Site Number : 840106 Birmingham, Alabama, United States
  • Barrow Neurological Institute- Site Number : 840087 Phoenix, Arizona, United States
  • Phoenix Children's Hospital- Site Number : 840003 Phoenix, Arizona, United States
  • University of Arizona- Site Number : 840015 Tucson, Arizona, United States
View all 272 locations
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Recruiting

A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

NCT07399704

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +23 more
Locations
  • Associação Hospitalar de Prot à Infância Dr. Raul Carneiro Água Verde, Curitiba, Brazil
  • Hospital de Clinicas de Porto Alegre Porto Alegre, Rio Grande do Sul, Brazil
  • Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira Rio de Janeiro, Brazil
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