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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

A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD )

NCT06559176

Conditions
Chronic Disease, Chronic Granulomatous Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +12 more
Locations
  • University of California Los Angeles Medical Center Los Angeles, California, United States
  • NIH Clinical Center Bethesda, Maryland, United States
  • The Children's Hospital at Tristar Medical Group/Sarah Cannon Center for Blood Cancers Nashville, Tennessee, United States
  • CHU - Sainte Justine Hospital Montreal, Quebec, Canada
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Enrolling by Invitation

Sirolimus for Leigh Syndrome

NCT06843811

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +12 more
Locations
  • Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
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Enrolling by Invitation

Long Term Follow-up for RGX-202

NCT06491927

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • Stanford School of Medicine /Division of Neuromuscular Medicine Palo Alto, California, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • The University of Texas Southwestern Medical Center Dallas, Texas, United States
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Enrolling by Invitation

Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy

NCT07209332

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations
  • Istiklal Hospital/ Clinical Research Unit Amman, Jordan
  • The Specialty Hospital (TSH)/ Advanced Clinical Center Amman, Jordan
  • Oxford Children's Hospital, Oxford University Hospitals NHS Foundation Trust Headington, Oxford, United Kingdom
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