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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

Enrolling by Invitation

A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study

NCT05967351

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
  • UCLA Medical Center Los Angeles, California, United States
  • Lucile Packard Children's Hospital Stanford (LPCH) Palo Alto, California, United States
  • University of California, Davis Sacramento, California, United States
View all 38 locations
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Enrolling by Invitation

An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects

NCT05594992

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +16 more
Locations
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
  • Hospital Universitario Austral Buenos Aires, Argentina
  • Hospital de Clínicas de Porto Alegre Porto Alegre, Brazil
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Enrolling by Invitation

A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice

NCT06606340

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations
  • Arkansas Children's Hospital Research Institute Little Rock, Arkansas, United States
  • Children's Hospital Los Angeles Los Angeles, California, United States
  • University of California Davis Medical Center Sacramento, California, United States
  • Children's National Hospital Washington D.C., District of Columbia, United States
View all 20 locations
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Enrolling by Invitation

Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)

NCT05962398

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +7 more
Locations
  • 10-15 Phoenix Childrens Hospital Phoenix, Arizona, United States
  • 10-14 Arkansas Children's Hospital - Pharmacology Little Rock, Arkansas, United States
  • 10-22 Orthopaedic Institute for Children Los Angeles, California, United States
  • 10-21 Children's Hospital of Los Angeles Los Angeles, California, United States
View all 28 locations
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