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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.

519 public trials 100 recruiting studies
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Lysosomal Storage Diseases, Nervous System trial results

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

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)

NCT07082725

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +20 more
Locations
  • UCSF Children's Hospital and Research Center at Oakland Oakland, California, United States
  • University of Minnesota Medical School Minneapolis, Minnesota, United States
  • Mayo Clinic Children's Center - PIN Rochester, Minnesota, United States
  • Children's Medical Center Dallas Dallas, Texas, United States
View all 34 locations
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Recruiting

A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

NCT07354724

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • University of California-Irvine Irvine, California, United States
  • Duke University School of Medicine - Early Phase Research Unit Durham, North Carolina, United States
  • The Lysosomal & Rare Disorders Research & Treatment Center Fairfax, Virginia, United States
  • Washington University School of Medicine St Louis, Missouri, United States
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