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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.

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

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 and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis

NCT07054515

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +25 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 Childrens Center - PIN Rochester, Minnesota, United States
  • Children's Medical Center Dallas Dallas, Texas, United States
View all 38 locations
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Recruiting

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)

NCT07282847

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
Locations
  • Barrow Neurological Institute Phoenix, Arizona, United States
  • University of California, Irvine (UCI) Irvine, California, United States
  • Stanford Neuroscience Health Center Palo Alto, California, United States
  • NYU Langone New York, 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 Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)

NCT05487599

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • Cedars-Sinai Los Angeles, California, United States
  • Ann and Robert H Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • Duke University Health System Durham, North Carolina, United States
  • Lysosomal & Rare Disorders Research and Treatment Center Fairfax, Virginia, United States
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