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Clinical trial condition

Rare Diseases

Explore clinical trials studying Rare Diseases. Study availability and eligibility vary by location and protocol.

466 public trials 83 recruiting studies
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Rare Diseases trial results

Recruiting

A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy

NCT07127978

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Duchene Muscular Dystrophy, +12 more
Locations
  • Arkansas Children's Research Institute Little Rock, Arkansas, United States
  • Children's National Hospital Washington D.C., District of Columbia, United States
  • Ann and Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • Boston Children's Hospital Boston, Massachusetts, United States
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Recruiting

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

NCT07123155

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +17 more
Locations
  • University of California - Irvine Medical Center Irvine, California, United States
  • University of Florida (UF) - Gainesville Gainesville, Florida, United States
  • Emory University Hospital Atlanta, Georgia, United States
  • Washington University in St. Louis St Louis, Missouri, United States
View all 28 locations
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Recruiting

A Study of SGT-212 Gene Therapy in Friedreich's Ataxia

NCT07180355

Conditions
Ataxia, Brain Diseases, Central Nervous System Diseases, +21 more
Locations
  • The University of California, Los Angeles (UCLA) Los Angeles, California, United States
  • The Ohio State University Columbus, Ohio, United States
  • The Children's Hospital of Philadelphia (CHOP) Philadelphia, Pennsylvania, United States
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