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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.

77 public trials 13 recruiting studies
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Lysosomal Storage Diseases trial results

Recruiting

A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II

NCT05208281

Conditions
Carbohydrate Metabolism, Inborn Errors, Cognition Disorders, Cognitive Dysfunction, +30 more
Locations
  • Federal State-Funded Healthcare Institution Central Clinical Hospital of the Russian Academy of Sciences (Research Institute of Pediatrics and Child Health Protection of the Central Clinical Hospital of the Russian Academy of Sciences) Moscow, Russia
  • Federal State Budgetary Educational Institution of Higher Education "St. Petersburg State Pediatric Medical University" of the Ministry of Health of the Russian Federation Saint Petersburg, Russia
  • V.I. Vernadsky Crimean Federal University Simferopol, Russia
  • State Budgetary Healthcare Institution Republican Medical Genetic Center Ufa, Russia
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Completed

Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)

NCT04046224

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • University of California, Irvine Irvine, California, United States
  • University of South Florida Tampa, Florida, United States
  • Emory University School of Medicine Atlanta, Georgia, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
View all 18 locations
View Trial Details

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