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Clinical trial condition

Genetic Diseases, X-Linked

Explore clinical trials studying Genetic Diseases, X-Linked. Study availability and eligibility vary by location and protocol.

1,243 public trials 227 recruiting studies
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Genetic Diseases, X-Linked trial results

Recruiting

A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease

NCT07187440

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • The First Affiliated Hospital Of USTC (AnHui Provincial Hospital) Hefei, Anhui, China
  • Peking University First Hospital Beijing, Beijing Municipality, China
  • Peking University Third Hospital Beijing, Beijing Municipality, China
  • Peking Union Medical College Hospital Beijing, Beijing Municipality, China
View all 18 locations
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Recruiting

Validating Innovative Biosensors for Rett Autonomic Symptom Tracking

NCT06338267

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +7 more
Locations
  • Rush University Medical Center Chicago, Illinois, United States
  • Boston Children's Hospital Boston, Massachusetts, United States
  • Baylor College of Medicine Houston, Texas, United States
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Recruiting

Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)

NCT04906460

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • Rare Disease Research LLC Atlanta, Georgia, United States
  • Istiklal Hospital/ Clinical Research Unit Amman, Jordan
  • The Specialty Hospital (TSH)/ Advanced Clinical Center Amman, Jordan
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Recruiting

Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)

NCT06150716

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +12 more
Locations
  • Ionis Investigative Site Atlanta, Georgia, United States
  • Ionis Investigative Site Philadelphia, Pennsylvania, United States
  • Ionis Investigative Site Salt Lake City, Utah, United States
  • Ionis Investigative Site Le Kremlin-Bicêtre, France
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Recruiting

Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning

NCT03311503

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Gene Therapy, Genetic Diseases, Inborn, +8 more
Locations
  • Mattel Children's Hospital - UCLA Los Angeles, California, United States
  • Emory University/Childrens Healthcare of Atlanta Atlanta, Georgia, United States
  • Boston Childrens Hospital Boston, Massachusetts, United States
  • Cincinnati Children's Hospital Medical Center Cincinnati, Ohio, United States
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