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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.

12,537 public trials 2,139 recruiting studies
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Genetic Diseases, Inborn trial results

Recruiting

Pediatric Evaluation and Registry for Liver Cholestasis in Canada

NCT07411716

Conditions
Abnormalities, Multiple, Alagille Syndrome, Alagille Syndrome (ALGS), +14 more
Locations
  • Alberta Children's Hospital Calgary, Alberta, Canada
  • Stollery Children's Hospital Edmonton, Alberta, Canada
  • BC Children's Hospital Vancouver, British Columbia, Canada
  • HSC Winnipeg Children's Hospital Winnipeg, Manitoba, Canada
View all 13 locations
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Recruiting

A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

NCT06092034

Conditions
Aortic Stenosis, Subvalvular, Aortic Valve Disease, Aortic Valve Stenosis, +26 more
Locations
  • University of California, San Diego La Jolla, California, United States
  • Boston Children's Hospital Boston, Massachusetts, United States
  • Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
  • German Heart Center Munich Munich, Germany
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Recruiting

Kidney Function in People With Cystic Fibrosis in the Era of HEMT

NCT06595420

Conditions
Acute Kidney Injury, Chronic Disease, Chronic Kidney Disease(CKD), +20 more
Locations
  • University of Alabama at Birmingham Birmingham, Alabama, United States
  • Dartmouth-Hitchcock Geisel School of Medicine at Dartmouth Lebanon, New Hampshire, United States
  • University of Virginia Children's Hospital Charlottesville, Virginia, United States
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Recruiting

External Multicenter Validation of the APTTO Model for Prolonged APTT Using Clot Waveform Analysis

NCT07418099

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Blood Platelet Disorders, +12 more
Locations
  • Hospital Universitario Severo Ochoa Leganés, Madrid, Spain
  • Hospital de la Santa Creu i Sant Pau Barcelona, Spain
  • Hospital Universitario Arnau De Vilanova Lleida, Spain
  • Hospital Universitario 12 de Octubre Madrid, Spain
View all 16 locations
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Recruiting

A Long-Term Study of JNT-517 in Participants With Phenylketonuria

NCT06628128

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +10 more
Locations
  • University of Florida (UF) Health Shands Hospital Gainesville, Florida, United States
  • University of South Florida Tampa, Florida, United States
  • Oregon Health and Science University Portland, Oregon, United States
  • University of Pittsburgh Medical Center (UPMC) - Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States
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Recruiting

A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy

NCT07047144

Conditions
Anti-myostatin, Central Nervous System Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +17 more
Locations
  • Phoenix Children's Hospital Phoenix, Arizona, United States
  • Children's Hospital of Orange County (CHOC) Orange, California, United States
  • Stanford Neuroscience Health Center (SNHC) Palo Alto, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
View all 25 locations
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