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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

A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy

NCT03771898

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Los Angeles Biomedical Research Institute at Harbor-UCLA Torrance, California, United States
  • Childrens Hospital Colorado Aurora, Colorado, United States
  • Rare Disease Research, LLC Atlanta, Georgia, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
View all 30 locations
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Completed

Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3

NCT03485677

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
Locations
  • Investigational Site Number : 0320001 Buenos Aires, Argentina
  • Investigational Site Number : 1240002 Calgary, Alberta, Canada
  • Investigational Site Number : 1240003 Vancouver, British Columbia, Canada
  • Investigational Site Number : 1240001 Toronto, Ontario, Canada
View all 21 locations
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Completed

A Gene Therapy Study in Patients With Gaucher Disease Type 1

NCT05324943

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • Kaiser Permanente Los Angeles, California, United States
  • Lysosomal Rare Disorders Research and Treatment Center Fairfax, Virginia, United States
  • Hospital de Clinicas de Porto Alegre (HCPA) Porto Alegre, Brazil
  • SphinCS Höchheim, Germany
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