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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.

710 public trials 130 recruiting studies
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Lysosomal Storage Diseases 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

Study of Skeletal Disorders

NCT05031507

Conditions
Bone Diseases, Bone Diseases, Developmental, Carbohydrate Metabolism, Inborn Errors, +15 more
Locations
  • National Institutes of Health Clinical Center Bethesda, Maryland, United States
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Recruiting

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

NCT02716246

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +12 more
Locations
  • Nationwide Children's Hospital Columbus, Ohio, United States
  • Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States
  • Women's and Children's Hospital North Adelaide, South Australia, Australia
  • Vall d'Hebron Barcelona Hospital Campus Barcelona, Spain
View Trial Details
Recruiting

Prospective Clinical Assessment Study in Children With Hypochondroplasia

NCT06410976

Conditions
Bone Diseases, Bone Diseases, Developmental, Carbohydrate Metabolism, Inborn Errors, +16 more
Locations
  • UCSF Benioff Children's Hospital Oakland, California, United States
  • Childrens Hospital Colorado Aurora, Colorado, United States
  • Children's National Hospital Washington D.C., District of Columbia, United States
  • Johns Hopkins School of Medicine Baltimore, Maryland, United States
View all 25 locations
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Recruiting

Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)

NCT07169279

Conditions
Achondroplasia, Bone Diseases, Bone Diseases, Developmental, +19 more
Locations
  • UCSF Benioff Children's Hospital Oakland, California, United States
  • Johns Hopkins University Baltimore, Maryland, United States
  • University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic Madison, Wisconsin, United States
  • Murdoch Children's Research Institute Parkville, Victoria, Australia
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