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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.

456 public trials 107 recruiting studies
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Genetic Diseases, Inborn trial results

Recruiting

A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study

NCT05130437

Conditions
Acid-Base Imbalance, Acidosis, Amino Acid Metabolism, Inborn Errors, +6 more
Locations
  • Ronald Reagan UCLA Medical Center Los Angeles, California, United States
  • University of Stanford Medical Center Palo Alto, California, United States
  • University of Michigan Hospitals Ann Arbor, Michigan, United States
  • Icahn School of Medicine at Mount Sinai New York, United States
View all 22 locations
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Recruiting

Helix Research Network

NCT06057181

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Disease Susceptibility, +5 more
Locations
  • Parkview Health (DNA Insights) Fort Wayne, Indiana, United States
  • HealthPartners (myGenetics) Bloomington, Minnesota, United States
  • Nebraska Medicine - University of Nebraska Medical Center (Genetic Insights Project) Omaha, Nebraska, United States
  • Renown Health (Healthy Nevada Project) Reno, Nevada, United States
View all 14 locations
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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

The China Neonatal Genomes Project

NCT03931707

Conditions
Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Defect, Congenital, +8 more
Locations
  • Children Hospital of Fudan University Shanghai, Shanghai Municipality, China
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Recruiting

PKP2-ACM Natural History Study

NCT06644742

Conditions
Arrhythmogenic Right Ventricular Dysplasia, Cardiomyopathies, Cardiovascular Abnormalities, +7 more
Locations
  • Boston Children's Hospital Boston, Massachusetts, United States
  • Duke University Durham, North Carolina, United States
  • Amsterdam UMC Amsterdam, Netherlands
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