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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

Cystadrops in Pediatric Cystinosis Patients From Six Months to Less Than Two Years Old (SCOB2)

NCT04125927

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystinosis, Genetic Diseases, Inborn, +4 more
Locations
  • UZ Leuven Leuven, Belgium
  • Hôpital Necker-Enfants Malades Paris, France
  • Klinik für Pädiatrische Nieren Hanover, Germany
  • Bambin Gesù Hospital in Palidoro Roma, Italy
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Completed

N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)

NCT03759639

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Mayo Clinic Rochester, Minnesota, United States
  • University of Giessen Giessen, Germany
  • Ludwig Maximilian University of Munich München, Germany
  • Comenius University in Bratislva Bratislava, Slovakia
View Trial Details
Completed

Global Burden and Treatment Trajectiories in Italian Patients With Fabry Disease

NCT04916977

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • AUSL Toscana Sud-Est - Ospedale San Donato Arezzo, Italy
  • A.O.U. Careggi Florence, Italy
  • U.O.C. Nefrologia ASST Monza - Ospedale San Gerardo Monza, Italy
  • U.O. di Nefrologia - Dipartimento di Sanità Pubblica Università degli Studi di Napoli "Federico II" NAPOLI Naples, Italy
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