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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 Cannabidiol in Sanfilippo Syndrome

NCT06333041

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations
  • The Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center Torrance, California, United States
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Recruiting

A Study of Patients With Fabry Disease (US Specific)

NCT06906367

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • UAB Nephrology Research Clinic at Paula Building Birmingham, Alabama, United States
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • Emory Genetics Atlanta, Georgia, United States
  • Washington University School of Medicine St Louis, Missouri, United States
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Recruiting

A Gaucher Disease Gene Therapy Trial With FLT201

NCT07223944

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • Kaiser Permanente - CA Los Angeles, California, United States
  • UCLA Health Los Angeles, California, United States
  • University of California, San Francisco (UCSF) San Francisco, California, United States
  • University of Colorado Anschutz Aurora, Colorado, United States
View all 33 locations
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Recruiting

A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants

NCT06904261

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Emory Genetics Atlanta, Georgia, United States
  • University of Minnesota Masonic Children's Hospital Minneapolis, Minnesota, United States
  • Atrium Health Levine Children's Hospital Charlotte, North Carolina, United States
  • Cincinnati Children's Hospital Medical Center Cincinnati, Ohio, United States
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Recruiting

Mucopolysaccharidosis VII Disease Monitoring Program

NCT03604835

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +12 more
Locations
  • Children's Hospital of Orange County Orange, California, United States
  • Children's National Health System Washington D.C., District of Columbia, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • University of Michigan Ann Arbor, Michigan, United States
View all 14 locations
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