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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.

12,522 public trials 2,142 recruiting studies
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Genetic Diseases, Inborn trial results

Recruiting

Extension Study for Participants in Studies That Include Belzutifan (MK-6482-043/LITESPARK-043)

NCT07405164

Conditions
Abnormalities, Multiple, Angiomatosis, Cardiovascular Diseases, +11 more
Locations
  • Beth Israel Deaconess Medical Center ( Site 0107) Boston, Massachusetts, United States
  • Dana-Farber Cancer Institute ( Site 0105) Boston, Massachusetts, United States
  • Karmanos Cancer Center ( Site 0108) Detroit, Michigan, United States
  • Hospital of the University of Pennsylvania Perelman Center for Advanced Medicine ( Site 0100) Philadelphia, Pennsylvania, United States
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Recruiting

A Study in Adults to Learn About Inherited Alpha-1 Antitrypsin Deficiency (AATD) and AATD Related Liver Problems

NCT06512454

Conditions
Alpha1-Antitrypsin Deficiency, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, +9 more
Locations
  • University of Florida Gainesville, Florida, United States
  • University of South Carolina Charleston, South Carolina, United States
  • Vanderbilt University Medical Center Nashville, Tennessee, United States
  • Vienna General Hospital (AKH Wien) Vienna, Austria
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Recruiting

Apremilast Pediatric Study in Children With Active Oral Ulcers Associated With Behçet's Disease

NCT04528082

Conditions
Behcet Syndrome, Behçet Disease, Cardiovascular Diseases, +17 more
Locations
  • Hospices Civils de Lyon Hopital Femme Mere Enfant Bron, France
  • Hopital Necker Enfants Malades Paris, France
  • Hopital Robert Debre Paris, France
  • Agia Sofia Children Hospital Athens, Greece
View all 27 locations
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Recruiting

A Study to Evaluate Atumelnant in Adults With Congenital Adrenal Hyperplasia

NCT07144163

Conditions
Adrenal Gland Diseases, Adrenal Hyperplasia, Congenital, Adrenogenital Syndrome, +17 more
Locations
  • Crinetics Study Site Los Angeles, California, United States
  • Crinetics Study Site Atlanta, Georgia, United States
  • Crinetics Study Site Chicago, Illinois, United States
  • Crinetics Study Site Baltimore, Maryland, United States
View all 55 locations
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Recruiting

Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy

NCT05933057

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations
  • Universitaire Ziekenhuizen Leuven Leuven, Belgium
  • British Columbia Children's Hospital Vancouver, British Columbia, Canada
  • The University of Western Ontario - Children's Health Research Institute London, Ontario, Canada
  • University of Ottawa - Children's Hospital of Eastern Ontario Ottawa, Ontario, Canada
View all 29 locations
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