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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

Mucopolysaccharidosis I (MPS I) Registry

NCT00144794

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations
  • Phoenix Children's Hospital- Site Number : 840003 Phoenix, Arizona, United States
  • Arkansas Children's Hospital- Site Number : 840109 Little Rock, Arkansas, United States
  • Southern California Permanente Medical Group- Site Number : 840108 Los Angeles, California, United States
  • USC Health Sciences Center Dept of Genetics- Site Number : 840082 Los Angeles, California, United States
View all 141 locations
View Trial Details
Completed

PREDIGA 2: Spanish Acronym of "Educational and Diagnostic Project for Gaucher and ASMD"

NCT05641103

Conditions
ASMD, Acid Sphingomyelinase Deficiency, Brain Diseases, +25 more
Locations
  • Hospital General Universitario de Alicante Alicante, Spain
  • Hospital de Poniente El Ejido, Almería, Spain
  • Hospital Universitario de Badajoz Badajoz, Spain
  • Hospital Vall d'Hebron Barcelona, Spain
View all 14 locations
View Trial Details
Completed

Switch Over Study of Biosimilar Agalsidase Beta for Fabry Disease

NCT05843916

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Instituto de Nefrología Pergamino S.R.L Pergamino, Buenos Aires, Argentina
  • Centro Médico Santa María de la Salud San Isidro, Buenos Aires, Argentina
  • Instituto de Investigaciones Clínicas Quilmes Buenos Aires, Argentina
  • Clínica Universitaria Reina Fabiola Córdoba, Argentina
View Trial Details
Completed

Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease

NCT04049760

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • University of South Florida Tampa, Florida, United States
  • The Emory Clinic Atlanta, Georgia, United States
  • University of Minnesota Masonic Children's Hospital and Clinics Minneapolis, Minnesota, United States
  • Cincinnati Children's Hospital Cincinnati, Ohio, United States
View Trial Details
Completed

A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.

NCT04281537

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Amicus Therapeutics, Inc. Philadelphia, Pennsylvania, United States
  • Instituto de Genética e Erros Inatos do Metabolismo (IGEIM) São Paulo, Brazil
  • Keio University Hospital Tokyo, Japan
  • Yokohama Municipal Citizen's Hospital Yokohama, Japan
View Trial Details

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