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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.

34 public trials 3 recruiting studies
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Genetic Diseases, Inborn trial results

Recruiting

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

NCT05477563

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +11 more
Locations
  • New York Presbyterian Hospital - Morgan Stanley Children's Hospital New York, United States
  • Levine Children's Hospital - Hematology Charlotte, North Carolina, United States
  • TriStar Medical Group Children's Specialists - Pediatric Oncology Nashville, Tennessee, United States
  • University Hospital Dusseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology Düsseldorf, Germany
View Trial Details
Recruiting

A Study to Learn About the Safety and Efficacy of the Drug Oleogel-S10 in Japanese Patients With Epidermolysis Bullosa

NCT06917690

Conditions
Collagen Diseases, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +13 more
Locations
  • Fukuoka Children's Hospital - Dermatology Fukuoka, Japan
  • Kurume University Hospital Fukuoka, Japan
  • Kobe University Hospital Kobe, Japan
  • Niigata University Medical and Dental Hospital Niigata, Japan
View Trial Details
Enrolling by Invitation

A Long-term Follow-up Study in Participants Who Received CTX001

NCT04208529

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +11 more
Locations
  • Lucile Packard Children's Hospital Palo Alto, California, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago - Hematology Chicago, Illinois, United States
  • Herbert Irving Pavilion - Hematology New York, United States
  • New York Presbyterian Hospital - Morgan Stanley Children's Hospital New York, United States
View all 20 locations
View Trial Details
Completed

Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

NCT03179631

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +8 more
Locations
  • Phoenix Childrens Hospital Phoenix, Arizona, United States
  • Children's Hospital of Los Angeles Los Angeles, California, United States
  • University of California, San Francisco (UCSF) - Benioff Children's Hospital - Oakland Oakland, California, United States
  • Stanford University Medical Center Palo Alto, California, United States
View all 64 locations
View Trial Details
Completed

A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia

NCT03655678

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +8 more
Locations
  • Lucile Packard Children's Hospital Palo Alto, California, United States
  • Ann & Robert Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • Columbia University Medical Center (21+ years) New York, United States
  • Columbia University Medical Center New York, United States
View all 14 locations
View Trial Details
Completed

Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients

NCT05479981

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DM1, Genetic Diseases, Inborn, +11 more
Locations
  • University of California Los Angeles Los Angeles, California, United States
  • Stanford University Palo Alto, California, United States
  • University of Colorado Denver, Colorado, United States
  • University of Florida Gainesville, Florida, United States
View Trial Details

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