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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

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

NCT05524883

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy (DMD), Genetic Diseases, Inborn, +8 more
Locations
  • University of California San Diego La Jolla, California, United States
  • UCLA University California of Los Angeles Los Angeles, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
  • Rare Disease Research, LLC Atlanta, Georgia, United States
View all 30 locations
View Trial Details
Active, Not Recruiting

A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome

NCT04251026

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +22 more
Locations
  • UCSF Benioff Children's Hospital Oakland, California, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • UNC Children's Research Institute Chapel Hill, North Carolina, United States
  • UPMC | Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States
View Trial Details
Active, Not Recruiting

An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II

NCT04348136

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +16 more
Locations
  • Fukui Clinical site Fukui, Japan
  • Fukuoka Clinical site 2 Fukuoka, Japan
  • Fukuoka Clinical site Fukuoka, Japan
  • Gifu Clinical site Gifu, Japan
View all 20 locations
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Active, Not Recruiting

Extension Study of NS-089/NCNP-02 in DMD

NCT05135663

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy (DMD), Genetic Diseases, Inborn, +8 more
Locations
  • Kagoshima University Hospital Kagoshima, Japan
  • National Center of Neurology and Psychiatry Tokyo, Japan
View Trial Details
Active, Not Recruiting

Arrhythmia Burden, Risk of Sudden Cardiac Death and Stroke in Patients With Fabry Disease

NCT03305250

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • University of Sydney Sydney, Australia
  • University Hospitals Birmingham NHS Foundation Trust Birmingham, West Midlands, United Kingdom
  • Cambridge University Hospitals NHS Foundation Trust Cambridge, United Kingdom
  • Cardiff and Vale University Health Board Cardiff, United Kingdom
View Trial Details
Active, Not Recruiting

Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:

NCT02616484

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
Locations
  • Children's Hospital of Orange County Orange, California, United States
  • Stanford University Stanford, California, United States
  • Children's National Medical Center Washington D.C., District of Columbia, United States
  • University of Florida Gainesville, Florida, United States
View Trial Details

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