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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.

449 public trials 107 recruiting studies
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Genetic Diseases, Inborn trial results

Completed

Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A

NCT04323098

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Cardiovascular Diseases, +9 more
Locations
  • University of California Davis Health Sacramento, California, United States
  • Washington University School of Medicine St Louis, Missouri, United States
  • Nationwide Children's Hospital Columbus, Ohio, United States
  • The Royal Adelaide Hospital Adelaide, Australia
View Trial Details
Completed

Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients

NCT05479981

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DM1, Genetic Diseases, Inborn, +11 more
Locations
  • University of California Los Angeles Los Angeles, California, United States
  • Stanford University Palo Alto, California, United States
  • University of Colorado Denver, Colorado, United States
  • University of Florida Gainesville, Florida, United States
View Trial Details
Completed

GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)

NCT05109793

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • UCSF Benioff Children's Hospital Oakland, California, United States
  • Mayo Clinic Rochester Rochester, Minnesota, United States
  • Hospital Pequeno Principe Curitiba, Brazil
  • Hospital de Clinicas de Porto Alegre Porto Alegre, Brazil
View all 13 locations
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Completed

An International Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B

NCT03901755

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +7 more
Locations
  • Swedish Orphan Biovitrum Research Site Brno, Czechia
  • Swedish Orphan Biovitrum Research Site Prague, Czechia
  • Swedish Orphan Biovitrum Research Site (Haemophilia Center Aghia Sophia Children's Hospital) Athens, Greece
  • Swedish Orphan Biovitrum Research Site (Laikο General Hospital of Athens) Athens, Greece
View all 29 locations
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