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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.

12,530 public trials 2,140 recruiting studies
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Genetic Diseases, Inborn trial results

Recruiting

REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome

NCT06914609

Conditions
Abnormalities, Multiple, Angelman Syndrome, Central Nervous System Diseases, +7 more
Locations
  • Cedars-Sinai Medical Center Los Angeles, California, United States
  • Rady Children's Hospital San Diego, California, United States
  • Colorado Children's Hospital Research Institute Aurora, Colorado, United States
  • Children's National Hospital Washington D.C., District of Columbia, United States
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Recruiting

Lentiviral FVIII Gene Therapy

NCT03217032

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +6 more
Locations
  • Shenzhen Geno-immune Medical Institute Shenzhen, Guangdong, China
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Recruiting

Chatbot to Maximize Hereditary Cancer Genetic Risk Assessment

NCT05562778

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Gynecologic Cancer, +3 more
Locations
  • NYP Brooklyn Methodist Hospital Brooklyn, New York, United States
  • NYP Lower Manhattan Hospital New York, United States
  • Weill Cornell Medicine New York, United States
  • NYP Medical Group Queens Queens, New York, United States
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Recruiting

Phase 1/2 Study of ETX-636 in Participants With Advanced Solid Tumors

NCT06993844

Conditions
Adnexal Diseases, Advanced Breast Cancer, Advanced Solid Tumors, +40 more
Locations
  • Hoag Memorial Hospital Presbyterian Newport Beach, California, United States
  • UCSF Helen Diller Family Comprehensive Cancer Center San Francisco, California, United States
  • Yale University, Yale Cancer Center New Haven, Connecticut, United States
  • Beth Israel Deaconess Medical Center Boston, Massachusetts, United States
View all 15 locations
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Recruiting

Fabry Disease Registry & Pregnancy Sub-registry

NCT00196742

Conditions
Angiokeratoma, Brain Diseases, Brain Diseases, Metabolic, +23 more
Locations
  • University of Alabama Birmingham- Nephrology- Site Number : 840018 Birmingham, Alabama, United States
  • University of Alabama Birmingham- Nephrology- Site Number : 840073 Birmingham, Alabama, United States
  • Phoenix Children's Hospital- Site Number : 840003 Phoenix, Arizona, United States
  • University of Arizona- Site Number : 840015 Tucson, Arizona, United States
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Recruiting

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

NCT07123155

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +17 more
Locations
  • University of California - Irvine Medical Center Irvine, California, United States
  • University of Florida (UF) - Gainesville Gainesville, Florida, United States
  • Emory University Hospital Atlanta, Georgia, United States
  • Washington University in St. Louis St Louis, Missouri, United States
View all 28 locations
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