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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.

12,603 public trials 2,158 recruiting studies
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Genetic Diseases, Inborn trial results

Active, not recruiting

An Extension Study to Evaluate the Long-Term Safety and Efficacy of Votoplam in Participants With Huntington's Disease (HD)

NCT06254482

Conditions
Basal Ganglia Diseases, Brain Diseases, Central Nervous System Diseases, +17 more
Locations
  • Novartis Investigative Site Clayton, Australia
  • Novartis Investigative Site Westmead, Australia
  • Novartis Investigative Site Innsbruck, Austria
  • Novartis Investigative Site Ottawa, Canada
View all 24 locations
View Trial Details
Active, not recruiting

A Clinical Study Evaluating the Safety and Efficacy of SKG0201 Injection in Patients With Spinal Muscular Atrophy Type 1

NCT06191354

Conditions
Central Nervous System Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +9 more
Locations
  • West China Sencond Hospital, Sichuan University / West China women's and children's Hospital Chengdu, Sichuan, China
  • Xinhua Hospital Affiliated To Shanghai Jiao Tong University School Of Medicine Shanghai, China
  • National Children's Medical Center, Children's Hospital of Fudan University Shanghai, China
View Trial Details
Active, not recruiting

Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.

NCT05145062

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +10 more
Locations
  • UCSF Benioff Children's Hospital Oakland, California, United States
  • University of California Davis Health System Sacramento, California, United States
  • Children's Healthcare of Atlanta Atlanta, Georgia, United States
  • Boston Children's Hospital Boston, Massachusetts, United States
View Trial Details
Active, not recruiting

A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)

NCT05540860

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
  • UC Davis Medical Center Sacramento, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
View all 14 locations
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Active, not recruiting

A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice

NCT04475926

Conditions
Cardiomyopathies, Cardiovascular Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +13 more
Locations
  • Barrow Neurological Institute Phoenix, Arizona, United States
  • Arkansas Children's Little Rock, Arkansas, United States
  • University of California San Diego La Jolla, California, United States
  • University of California, Davis Health Dept of PM&R Sacramento, California, United States
View all 26 locations
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Active, not recruiting

Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy

NCT06100887

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations
  • UCLA Medical Center Los Angeles, California, United States
  • UC Davis Medical Center Sacramento, California, United States
  • University of Florida Gainesville, Florida, United States
  • University of Massachusetts Memorial Medical Center Worcester, Massachusetts, United States
View Trial Details
Active, not recruiting

Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment

NCT06031259

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +17 more
Locations
  • M.A.G.I.C. Clinic Calgary, Alberta, Canada
  • University of British Columbia Vancouver, British Columbia, Canada
  • Hospital for Sick Children Toronto, Ontario, Canada
  • Hopital Femme Mere Enfant Bron, Auvergne-Rhône-Alpes, France
View Trial Details

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