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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.

12,603 public trials 2,158 recruiting studies
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Genetic Diseases, Inborn trial results

Active, not recruiting

A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia

NCT05662319

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +7 more
Locations
  • Center for Inherited Blood Disorders (CIBD) Site Number : 8400012 Orange, California, United States
  • M Health Fairview University of Minnesota Medical Center - West Bank- Site Number : 8400016 Minneapolis, Minnesota, United States
  • Hackensack University Site Number : 8400009 Hackensack, New Jersey, United States
  • Northwell Health Hemostasis and Thrombosis Center Site Number : 8400015 New Hyde Park, New York, United States
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Active, not recruiting

A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease

NCT05206773

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Nephrology Clinic at Kirklin Clinic of UAB Hospital_Investigational Site Number: 8400011 Birmingham, Alabama, United States
  • UCLA Medical Center_Investigational Site Number: 8400006 Los Angeles, California, United States
  • University of California Irvine Medical Center- Site Number : 8400019 Orange, California, United States
  • Advent Health Orlando_Investigational Site Number: 8400008 Orlando, Florida, United States
View all 58 locations
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Active, not recruiting

Rate of Progression of PCDH15-Related Retinal Degeneration in Usher Syndrome 1F

NCT04765345

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Deafness, Autosomal Recessive 23, Eye Diseases, +6 more
Locations
  • University of California, San Francisco San Francisco, California, United States
  • The Johns Hopkins Wilmer Eye Institute Baltimore, Maryland, United States
  • Duke University, Duke Eye Center Durham, North Carolina, United States
  • Hospital for Sick Children Toronto, Ontario, Canada
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Active, not recruiting

A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease

NCT04537377

Conditions
Basal Ganglia Diseases, Brain Diseases, Brain Diseases, Metabolic, +16 more
Locations
  • UC Davis Medical Center Sacramento, California, United States
  • Yale University School of Medecine New Haven, Connecticut, United States
  • Advent Health Orlando, Florida, United States
  • University of Michigan Health System Ann Arbor, Michigan, United States
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