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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.

77 public trials 13 recruiting studies
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Lysosomal Storage Diseases trial results

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

Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting

NCT07109375

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • AUSL Toscana Sud-Est - Ospedale San Donato Arezzo, Italy
  • Azienda Ospedaliera Universitaria Consorziale Policlinico Di Bari Bari, Italy
  • Irccs Azienda Ospedaliero-Universitaria Di Bologna - Policlinico Di Sant'Orsola Bologna, Italy
  • A.O. Sant'Anna e San Sebastiano Caserta, Italy
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Recruiting

DFT383 in Pediatric Participants With Nephropathic Cystinosis

NCT06910813

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystinosis, Fanconi Syndrome, +13 more
Locations
  • University of California at San Diego - Rady Children's Hospital San Diego, California, United States
  • Stanford University - Stanford Children's Health Stanford, California, United States
  • Emory University School of Medicine - Children's Healthcare of Atlanta (recuiting Cohort 0) Atlanta, Georgia, United States
  • Baylor College of Medicine - Texas Children's Hospital (recuiting Cohort 0) Houston, Texas, United States
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Recruiting

A Long-Term Observational Study of Patients With Fucosidosis

NCT07615400

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +11 more
Locations
  • Children's Hospital of Orange County Orange, California, United States
  • University of Minnesota Health Minneapolis, Minnesota, United States
  • Indira Gandhi Institute of Child Health, Department of Pediatric Neurology Bengaluru, India
  • Erasmus University Medical Center Rotterdam, Netherlands
View all 16 locations
View Trial Details
Recruiting

A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

NCT06092034

Conditions
Aortic Stenosis, Subvalvular, Aortic Valve Disease, Aortic Valve Stenosis, +26 more
Locations
  • University of California, San Diego La Jolla, California, United States
  • Boston Children's Hospital Boston, Massachusetts, United States
  • Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
  • German Heart Center Munich Munich, Germany
View Trial Details

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