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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.

12,578 public trials 2,158 recruiting studies
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Genetic Diseases, Inborn trial results

Enrolling by Invitation

TANDem-2: Closing the Gap to Interventions for TAND

NCT06879665

Conditions
Adult Caregivers of Individuals With TSC, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +14 more
Locations
  • TSC Alliance Silver Spring, Maryland, United States
  • Tuberous Sclerosis Australia Beverly Hills, New South Wales, Australia
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Enrolling by Invitation

A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice

NCT06606340

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations
  • Arkansas Children's Hospital Research Institute Little Rock, Arkansas, United States
  • Children's Hospital Los Angeles Los Angeles, California, United States
  • University of California Davis Medical Center Sacramento, California, United States
  • Children's National Hospital Washington D.C., District of Columbia, United States
View all 20 locations
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Enrolling by Invitation

The BENeFiTS Trial in Beta Thalassemia Intermedia

NCT04432623

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +10 more
Locations
  • UCSF Benioff Children's Hospital at Oakland Oakland, California, United States
  • Massachusetts General Hospital Boston, Massachusetts, United States
  • Susan Perrine Weston, Massachusetts, United States
  • Weil Cornell Medicine New York, United States
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