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Clinical trial condition

Lysosomal Storage Diseases, Nervous System

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

513 public trials 105 recruiting studies
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Lysosomal Storage Diseases, Nervous System trial results

Completed

Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)

NCT03500094

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Clinical Study Site Tampa, Florida, United States
  • Clinical Study Site Atlanta, Georgia, United States
  • Clinical Study Site Minneapolis, Minnesota, United States
  • Clinical Study Site Columbia, Missouri, United States
View Trial Details
Completed

Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2

NCT04470713

Conditions
AB Variant Gangliosidosis GM2, Brain Diseases, Brain Diseases, Metabolic, +24 more
Locations
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • Mayo Clinic - Rochester Rochester, Minnesota, United States
  • Lysosomal and Rare Disorders Research and Treatment Center Fairfax, Virginia, United States
  • UCL Cliniques Universitaires Saint-Luc Brussels, Belgium
View all 17 locations
View Trial Details
Completed

Replagal Enzyme Replacement Therapy for Children With Fabry Disease

NCT00084084

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +20 more
Locations
  • Tucson Access Center of Arizona Kidney Disease Hypertension Center Tucson, Arizona, United States
  • University of Arizona Health Sciences Center Tucson, Arizona, United States
  • Children's Physician Group Palm Beach Gardens, Florida, United States
  • Christus St. Patrick Hospital Lake Charles, Louisiana, United States
View all 14 locations
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