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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

Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function

NCT02795676

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +29 more
Locations
  • UAB Medicine Birmingham, Alabama, United States
  • Phoenix Children's Hospital Phoenix, Arizona, United States
  • University of California Irvine Center Orange, California, United States
  • University of California San Diego San Diego, California, United States
View all 29 locations
View Trial Details
Completed

Extension Study of PRX-102 for up to 60 Months

NCT01981720

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • University of Florida Gainesville, Florida, United States
  • University of Iowa Hospitals and Clinics Iowa City, Iowa, United States
  • Johns Hopkins University School of Medicine Baltimore, Maryland, United States
  • Institute of Metabolic Disease Dallas, Texas, United States
View Trial Details
Completed

Genetic Newborn Screening for Cystinosis and Spinal Muscular Atrophy

NCT06027385

Conditions
Central Nervous System Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystinosis, +13 more
Locations
  • RoMed Hospital Rosenheim, Bavaria, Germany
  • University Hospital Essen, Center for Pediatrics and Adolescent Medicine Essen, Germany
  • Dr. von Haunersches Kinderspital München, Germany
  • University Hospital Münster, Clinic and Polyclinic for Pediatrics and Adolescent Medicine Münster, Germany
View Trial Details
Completed

A Prospective and Retrospective Cohort Study in Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD)

NCT04106544

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +20 more
Locations
  • Investigational Site Number :8400002 Atlanta, Georgia, United States
  • Investigational Site Number :8400003 The Bronx, New York, United States
  • Investigational Site Number :8400001 Valhalla, New York, United States
  • Investigational Site Number :0320002 CABA, Argentina
View all 28 locations
View Trial Details
Completed

Lamazym Aftercare Study

NCT01908725

Conditions
Alpha-Mannosidosis, Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +7 more
Locations
  • Center for Metabolic Diseases, Department of Clinical Genetics, Juliane Marie Centre, Copenhagen University Hospital, Blegdamsvej 9 Copenhagen, Denmark
View Trial Details

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