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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.

1,242 public trials 227 recruiting studies
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Genetic Diseases, X-Linked trial results

Active, Not Recruiting

A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)

NCT06450639

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Fractures, Bone, +13 more
Locations
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • University of California Davis Medical Center Sacramento, California, United States
  • Children's Healthcare of Atlanta Center for Advanced Pediatrics Atlanta, Georgia, United States
  • Corewell Health Grand Rapids, Michigan, United States
View all 19 locations
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Active, Not Recruiting

Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B

NCT03307980

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +8 more
Locations
  • UC Davis Comprehensive Cancer Center Sacramento, California, United States
  • UC Davis Ellison Ambulatory Care Clinic Sacramento, California, United States
  • UC Davis Medical Center department of Radiology Sacramento, California, United States
  • UC Davis Medical Center Sacramento, California, United States
View all 25 locations
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Active, Not Recruiting

A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD)

NCT06128564

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations
  • Chr de La Citadelle Liège, Belgium
  • Hôpital Necker-Enfants Malades Paris, France
  • Universitätsklinikum Essen Essen, Germany
  • PU A. Gemelli, Università Cattolica del Sacro Cuore Rome, Lazio, Italy
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Active, Not Recruiting

German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa

NCT06095713

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Fabry disease center Berlin - Charité - Universitätsmedizin Berlin Berlin, Germany
  • Fabry disease center Cologne, Universitätsklinikum Köln Cologne, Germany
  • Fabry disease center Hamburg, Universitätsklinikum Hamburg Hamburg, Germany
  • Fabry disease center Hannover, Universitätsklinikum Hannover Hanover, Germany
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Active, Not Recruiting

Liver Disease in Urea Cycle Disorders

NCT04612764

Conditions
ARGI Deficiency, ASL Deficiency, ASS Deficiency, +22 more
Locations
  • Children's Hospital Colorado Aurora, Colorado, United States
  • Children's National Medical Center Washington D.C., District of Columbia, United States
  • The Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
  • Baylor College of Medicine Houston, Texas, United States
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Active, Not Recruiting

A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy

NCT06713135

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations
  • UZ Gent (Universitair Ziekenhuis Gent) Ghent, Belgium
  • UZ Leuven (Universitair Ziekenhuis Leuven) Leuven, Belgium
  • University Hospital Brno Brno, Czechia
  • Fakultni Nemocnice Motol Prague, Czechia
View all 18 locations
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