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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

Emicizumab for Severe Von Willebrand Disease (VWD) and VWD/Hemophilia A

NCT05500807

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +10 more
Locations
  • The Center for Comprehensive Care and Diagnosis of Inherited Blood Disorders (CIBD) Orange, California, United States
  • Stanford University: Stanford Children's Health Redwood City, California, United States
  • University of Miami - Miller School of Medicine Coral Gables, Florida, United States
  • St. Joseph's Children's Hospital - Center for Bleeding and Clotting Disorders Tampa, Florida, United States
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Recruiting

Study to Evaluate Subcutaneous (SC) VGA039 in Patients With Von Willebrand Disease (VWD)

NCT07115004

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Blood Platelet Disorders, +8 more
Locations
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • Luskin Orthopaedic Institute For Children Los Angeles, California, United States
  • UC Davis Medical Center Sacramento, California, United States
  • University of California San Francisco San Francisco, California, United States
View all 14 locations
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Recruiting

A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

NCT05163288

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Mayo Clinic Rochester, Minnesota, United States
  • The Royal Melbourne Hospital Parkville, Victoria, Australia
  • First Faculty of Medicine, Charles University Hospital Prague Prague, Czechia
  • University of Giessen Giessen, Germany
View all 14 locations
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Recruiting

The Belgian Endothelial Surgical Transplant of the Cornea

NCT05436665

Conditions
Bullous Keratopathy, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Corneal Diseases, +8 more
Locations
  • Antwerp University Hospital Edegem, Antwerp, Belgium
  • AZ Maria Middelares Ghent, Oost-Vlaanderen, Belgium
  • AZ Sint-Jan Brugge Bruges, West-Vlaanderen, Belgium
  • AZ Imelda Bonheiden, Belgium
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