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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.

12,613 public trials 2,162 recruiting studies
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Genetic Diseases, Inborn trial results

Active, not recruiting

Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A

NCT03432520

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +6 more
Locations
  • Boston Children's Hospital Boston, Massachusetts, United States
  • Mississippi Center for Advanced Medicine Madison, Mississippi, United States
  • Truman Medical Centers Kansas City, Missouri, United States
  • Oregon Health & Science University Portland, Oregon, United States
View Trial Details
Active, not recruiting

A Natural History Study to TRACK Brain and Spinal Cord Changes in Individuals with Friedreich Ataxia (TRACK-FA)

NCT04349514

Conditions
Brain Diseases, Central Nervous System Diseases, Cerebellar Diseases, +11 more
Locations
  • University of Florida Gainesville, Florida, United States
  • Center for Magnetic Resonance Research, University of Minnesota Minneapolis, Minnesota, United States
  • Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
  • Monash Biomedical Imaging, Monash University Clayton, Victoria, Australia
View Trial Details
Active, not recruiting

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

NCT04093349

Conditions
Acid Maltase Deficiency, Brain Diseases, Brain Diseases, Metabolic, +17 more
Locations
  • Barrow Neurological Institute Phoenix, Arizona, United States
  • University of California Irvine Health Orange, California, United States
  • Emory University School of Medicine Atlanta, Georgia, United States
  • University of Kansas Medical Center Research Institute Kansas City, Kansas, United States
View all 29 locations
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Active, not recruiting

Early Human Leukocyte Antigen (HLA) Matched Sibling Hematopoietic Stem Cell Transplantation

NCT04018937

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations
  • Children's Hospital of Alabama Birmingham, Alabama, United States
  • Phoenix Children's Phoenix, Arizona, United States
  • Yale University, Yale Cancer Center New Haven, Connecticut, United States
  • Children's National Hospital Washington D.C., District of Columbia, United States
View all 15 locations
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Active, not recruiting

A Study of NDI 1150-101 in Patients With Solid Tumors

NCT05128487

Conditions
Adenocarcinoma, Bronchial Neoplasms, Carcinoma, +36 more
Locations
  • Honor Health Research Institute Scottsdale, Arizona, United States
  • Yale Cancer Center New Haven, Connecticut, United States
  • Georgetown University Washington D.C., District of Columbia, United States
  • Ocala Oncology Center Ocala, Florida, United States
View all 19 locations
View Trial Details
Active, not recruiting

Spinraza in Adult Spinal Muscular Atrophy

NCT03709784

Conditions
Central Nervous System Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +11 more
Locations
  • Barrow Neurological Institute Phoenix, Arizona, United States
  • Georgetown University Washington D.C., District of Columbia, United States
  • Johns Hopkins Baltimore, Maryland, United States
  • Massachusetts General Hospital-Harvard University Boston, Massachusetts, United States
View Trial Details

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