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Clinical trial condition

Genetic Diseases, Inborn

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

12,520 public trials 2,145 recruiting studies
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Genetic Diseases, Inborn trial results

Recruiting

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis

NCT07054515

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +25 more
Locations
  • UCSF Children's Hospital and Research Center at Oakland Oakland, California, United States
  • University of Minnesota Medical School Minneapolis, Minnesota, United States
  • Mayo Clinic Childrens Center - PIN Rochester, Minnesota, United States
  • Children's Medical Center Dallas Dallas, Texas, United States
View all 38 locations
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Recruiting

NIAID Centralized Sequencing Protocol

NCT03206099

Conditions
Atopy, Autoimmune Diseases, Autoimmunity, +7 more
Locations
  • Children's National Health System Washington D.C., District of Columbia, United States
  • National Institutes of Health Clinical Center Bethesda, Maryland, United States
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Recruiting

A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)

NCT06817382

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations
  • USA012 Little Rock, Arkansas, United States
  • USA010 Davis, California, United States
  • USA009 Los Angeles, California, United States
  • USA002 Palo Alto, California, United States
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Recruiting

Modeling Mortality in Duchenne Muscular Dystrophy Cardiomyopathy: Identification of Surrogate Outcome Measures for DMD Drug Trials

NCT07674758

Conditions
Becker Muscular Dystrophy, Cardiomyopathies, Cardiomyopathy, +14 more
Locations
  • UC Davis Sacramento, California, United States
  • Children's National Washington D.C., District of Columbia, United States
  • Lurie Children's Chicago, Illinois, United States
  • Riley Children's Hospital Indianapolis, Indiana, United States
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Recruiting

Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders

NCT07221851

Conditions
Bone Diseases, Bone Diseases, Developmental, Cardiovascular Abnormalities, +32 more
Locations
  • Ascendis Pharma Investigational Site Palo Alto, California, United States
  • Ascendis Pharma Investigational Site Sacramento, California, United States
  • Ascendis Pharma Investigational Site Aurora, Colorado, United States
  • Ascendis Pharma Investigational Site Centennial, Colorado, United States
View all 34 locations
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