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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

Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia

NCT06455059

Conditions
Bone Diseases, Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +6 more
Locations
  • Nemours Alfred I. DuPont Hospital for Children Wilmington, Delaware, United States
  • Children's National Medical Center Washington D.C., District of Columbia, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • Medical College of Wisconsin Milwaukee, Wisconsin, United States
View all 23 locations
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Active, Not Recruiting

Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease

NCT05297812

Conditions
Alpha 1-Antitrypsin Deficiency, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, +10 more
Locations
  • University of Alabama at Birmingham Birmingham, Alabama, United States
  • University of California - Los Angeles Los Angeles, California, United States
  • National Jewish Health Denver, Colorado, United States
  • University of Chicago Chicago, Illinois, United States
View Trial Details
Active, Not Recruiting

A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a)

NCT05095727

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +6 more
Locations
  • University of Connecticut Health Center Farmington, Connecticut, United States
  • Boston Children's Hospital Boston, Massachusetts, United States
  • Duke University Medical Center Durham, North Carolina, United States
  • Cincinnati Children's Hospital Medical Center Cincinnati, Ohio, United States
View all 16 locations
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Active, Not Recruiting

Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT

NCT05739643

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +20 more
Locations
  • Children's Hospital of Orange County (CHOC) Orange, California, United States
  • University of Michigan Hospitals - Michigan Medicine Ann Arbor, Michigan, United States
  • Duke University Medical Center Durham, North Carolina, United States
View Trial Details

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