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Clinical trial condition

Genetic Diseases, Inborn

Explore clinical trials studying Genetic Diseases, Inborn. Study availability and eligibility vary by location and protocol.

12,537 public trials 2,139 recruiting studies
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Genetic Diseases, Inborn trial results

Recruiting

PASS of Xromi Comparing Safety and Effectiveness in Children Under 2 Years With Sickle Cell Disease [PRECISE PASS]

NCT06923111

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations
  • Universitätsklinikum Hamburg-Eppendorf Hamburg, Germany
  • Universitätsklinikum Heidelberg Heidelberg, Germany
  • Basildon University Hospital Basildon, United Kingdom
  • Noah's Ark Children's Hospital for Wales Cardiff, United Kingdom
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Recruiting

Study to Learn About the Safety of Fazirsiran and if it Can Help People With Alpha-1 Antitrypsin Liver Disease With Mild Liver Scarring (Fibrosis)

NCT06165341

Conditions
Alpha1-Antitrypsin Deficiency, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, +9 more
Locations
  • St Joseph's Hospital and Medical Center Phoenix, Arizona, United States
  • Mayo Clinic - PPDS Phoenix, Arizona, United States
  • University of Arizona Thomas D. Boyer Liver Institute Tucson, Arizona, United States
  • University of California San Diego La Jolla, California, United States
View all 41 locations
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Recruiting

Trial of Cell Based Therapy for DMD

NCT06692426

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations
  • Masonic Cancer Center Minneapolis, Minnesota, United States
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