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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

Completed

Efficacy and Safety of mAnnitol in Bowel Preparation During Elective Colonoscopy and Comparison With Moviprep®

NCT04759885

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations
  • Centre Hospitalier Henri Duffaut Avignon, France
  • Hospices civils de Lyon Lyon, France
  • Hôpital Edouard Herriot Lyon, France
  • Centre Hospitalier Universitaire de Montpellier Montpellier, France
View all 35 locations
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Completed

Neuromuscular Characterisation in Late Adolescent and Adult Cystinosis Patients

NCT05545774

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystinosis, Deglutition Disorders, +17 more
Locations
  • Neurology Department, Raymond-Poincaré hospital, APHP Garches, France
  • Department of Nephrology and Transplantation Centre de référence des Maladies Rénales Héréditaires de l'Enfant et de l'Adulte, Necker-Enfants Malades University Hospital, APHP Paris, France
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Completed

First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

NCT02675465

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • Neuromuscular Research Centre Phoenix, Arizona, United States
  • University of California Irvine Orange, California, United States
  • University of Florida Gainesville, Florida, United States
  • Emory University Division of Medical Genetics Decatur, Georgia, United States
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Completed

Study of Infigratinib in Children With Achondroplasia

NCT04265651

Conditions
Achondroplasia, Bone Diseases, Bone Diseases, Developmental, +16 more
Locations
  • UCSF Benioff Children's Hospital Oakland, California, United States
  • Nemours Alfred I. Dupont Hospital for Children Wilmington, Delaware, United States
  • Johns Hopkins School of Medicine Baltimore, Maryland, United States
  • Cincinnati Children's Hospital Medical Center Cincinnati, Ohio, United States
View all 19 locations
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