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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.

12,530 public trials 2,140 recruiting studies
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Genetic Diseases, Inborn trial results

Recruiting

Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)

NCT07169279

Conditions
Achondroplasia, Bone Diseases, Bone Diseases, Developmental, +19 more
Locations
  • UCSF Benioff Children's Hospital Oakland, California, United States
  • Johns Hopkins University Baltimore, Maryland, United States
  • University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic Madison, Wisconsin, United States
  • Murdoch Children's Research Institute Parkville, Victoria, Australia
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Recruiting

Study to Evaluate the Pharmacodynamics, Safety and Efficacy of SKY-0515 in Participants With Huntington's Disease

NCT07378644

Conditions
Basal Ganglia Diseases, Brain Diseases, Central Nervous System Diseases, +13 more
Locations
  • IADIN (Instituto Argentino de Investigación Neurológica) Buenos Aires, Argentina
  • Buenos Aires British Hospital CABA, Buenos Aires, Argentina
  • Instituto Argentino de Investigación Neurológica CABA, Buenos Aires, Argentina
  • CINME Centro de Investigaciones Metabólicas CABA, Buenos Aires, Argentina
View all 19 locations
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Recruiting

ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders

NCT04398628

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +34 more
Locations
  • Arizona Hemophilia and Thrombosis Treatment Center at Phoenix Children's Hospital Phoenix, Arizona, United States
  • Arkansas Center for Bleeding Disorders Little Rock, Arkansas, United States
  • Orthopaedic Institute for Children HTC Los Angeles, California, United States
  • Childrens Hospital Los Angeles Los Angeles, California, United States
View all 71 locations
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Recruiting

A Gaucher Disease Gene Therapy Trial With FLT201

NCT07223944

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • Kaiser Permanente - CA Los Angeles, California, United States
  • UCLA Health Los Angeles, California, United States
  • University of California, San Francisco (UCSF) San Francisco, California, United States
  • University of Colorado Anschutz Aurora, Colorado, United States
View all 33 locations
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