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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

Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE

NCT05492799

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • UPMC Children's Hospital Pittburgh Pittsburgh, Pennsylvania, United States
  • Fundación Cardio Infantil - Instituto de Cardiología Bogotá, Colombia
  • University Medical Center Hamburg-Eppendorf Hamburg, Germany
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Enrolling by Invitation

Natural History Study of SCID Disorders

NCT01186913

Conditions
ADA SCID, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA Repair-Deficiency Disorders, +17 more
Locations
  • University of Alabama at Birmingham Birmingham, Alabama, United States
  • Phoenix Children's Hospital Phoenix, Arizona, United States
  • Children's Hospital Los Angeles Los Angeles, California, United States
  • University of California, Los Angeles Los Angeles, California, United States
View all 44 locations
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Not Yet Recruiting

Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)

NCT07681713

Conditions
Brain Diseases, Central Nervous System Diseases, Cerebellar Diseases, +12 more
Locations
  • University of California Los Angeles Los Angeles, California, United States
  • University of South Florida Tampa, Florida, United States
  • University of Iowa Iowa City, Iowa, United States
  • Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
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