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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

An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy

NCT06270719

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
  • Children's Hospital Los Angeles - PIN Los Angeles, California, United States
  • University of Colorado - PPDS Aurora, Colorado, United States
  • Connecticut Children's Medical Center - Hartford Hartford, Connecticut, United States
View all 25 locations
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Enrolling by Invitation

Li-Fraumeni Syndrome Imaging Study

NCT03176836

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA Repair-Deficiency Disorders, Genetic Diseases, Inborn, +5 more
Locations
  • The Hospital for Sick Children Toronto, Ontario, Canada
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Enrolling by Invitation

Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older

NCT05844449

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Digestive System Diseases, +5 more
Locations
  • Children's Hospital of Orange County Orange, California, United States
  • Stanford University - Palo Alto - Pulmonology Palo Alto, California, United States
  • Children's Hospital of Colorado - Pulmonology Aurora, Colorado, United States
  • The Emory Clinic - Clifton Road - Pulmonology Atlanta, Georgia, United States
View all 37 locations
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Enrolling by Invitation

Long Term Follow-Up for RGX-111

NCT06103487

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +9 more
Locations
  • Children's Hospital of Orange County Orange, California, United States
  • Hospital de Clinicas de Porto Alegre Porto Alegre, Rio Grande do Sul, Brazil
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Enrolling by Invitation

Defining the Skin and Blood Biomarkers of Ichthyosis

NCT03417856

Conditions
Abnormalities, Multiple, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +10 more
Locations
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • Northbrook Lurie Children's Outpatient Clinic Chicago, Illinois, United States
  • Northwestern University Chicago, Illinois, United States
  • Icahn School of Medicine at Mount Sinai New York, United States
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