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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

Active, Not Recruiting

The Electronic Medical Records and GEnomics (eMERGE) Network Genomic Risk Assessment

NCT05277116

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Disease, Genetic Diseases, Inborn
Locations
  • University of Alabama Birmingham Birmingham, Alabama, United States
  • Mountain Park Health Center Tempe, Arizona, United States
  • Northwestern Chicago, Illinois, United States
  • Massachusetts General Hospital Boston, Massachusetts, United States
View Trial Details
Active, Not Recruiting

Long Term Extension Study in Patients With Primary Hyperoxaluria

NCT04042402

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, +17 more
Locations
  • Clinical Research Site San Francisco, California, United States
  • Clinical Trial Site Boston, Massachusetts, United States
  • Clinical Trial Site Rochester, Minnesota, United States
  • Clinical Trial Site New York, United States
View all 23 locations
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Active, Not Recruiting

A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)

NCT04853576

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations
  • UCSF Benioff Children's Hospital Oakland, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
  • Smilow Cancer Hospital New Haven, Connecticut, United States
  • Johns Hopkins All Children's Hospital St. Petersburg, Florida, United States
View all 24 locations
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Active, Not Recruiting

An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease

NCT04519749

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • University of California at San Diego La Jolla, California, United States
  • Emory University Atlanta, Georgia, United States
  • Children's Hospital of Pittsburgh of UPMC Pittsburgh, Pennsylvania, United States
  • Lysosomal & Rare Disorders Research & Treatment Center, Inc Fairfax, Virginia, United States
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