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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

CNKSR2 Natural History Study

NCT06500260

Conditions
Brain Diseases, Central Nervous System Diseases, Communication Disorders, +15 more
Locations
  • University of California, San Francisco (UCSF) San Francisco, California, United States
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Active, Not Recruiting

Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

NCT04281485

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
  • Arkansas Children's Little Rock, Arkansas, United States
  • UCLA Medical Center Los Angeles, California, United States
  • University of Florida Gainesville, Florida, United States
View all 53 locations
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Active, Not Recruiting

AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)

NCT05693142

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
  • Children's Hospital of Orange County Orange, California, United States
  • Stanford School of Medicine /Division of Neuromuscular Medicine Palo Alto, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
View all 23 locations
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Active, Not Recruiting

Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials

NCT05335876

Conditions
Atrophy, Bulbo-Spinal Atrophy, X-Linked, Central Nervous System Diseases, +19 more
Locations
  • Child Hosp Of The Kings Daughters Norfolk, Virginia, United States
  • Novartis Investigative Site Sydney, New South Wales, Australia
  • Novartis Investigative Site Leuven, Belgium
  • Novartis Investigative Site Curitiba, Paraná, Brazil
View all 32 locations
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Active, Not Recruiting

A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease

NCT04020055

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +32 more
Locations
  • Emory University Atlanta, Georgia, United States
  • The Cleveland Clinic Cleveland, Ohio, United States
  • Lysosomal and Rare Disorders Research and Treatment Center, Inc Fairfax, Virginia, United States
  • Royal Melbourne Hospital Parkville, Victoria, Australia
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