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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.

12,597 public trials 2,159 recruiting studies
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Genetic Diseases, Inborn trial results

Active, not recruiting

Cardiopulmonary Outcomes in Osteogenesis Imperfecta: BBD7708

NCT05317637

Conditions
Bone Diseases, Bone Diseases, Developmental, Collagen Diseases, +7 more
Locations
  • University of California Los Angeles Los Angeles, California, United States
  • Kennedy Krieger Institute / Hugo W. Moser Research Institute Baltimore, Maryland, United States
  • Hospital for Special Surgery New York, United States
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Active, not recruiting

Dose-escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis Pigmentosa

NCT03326336

Conditions
Cone-Rod Dystrophies, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, +7 more
Locations
  • UPMC Eye Center Pittsburgh, Pennsylvania, United States
  • Centre Hospitalier National d'Ophtalmologie (CHNP) des Quinze-Vingts Paris, France
  • Moorfields Eye Hospital NHS Foundation Trust, 162 City Road London, United Kingdom
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Active, not recruiting

A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4

NCT06246513

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Limb-girdle Muscular Dystrophy, +7 more
Locations
  • University of California, San Diego-Altman Clinical and Translational Research Institute La Jolla, California, United States
  • Nationwide Childrens Hospital Columbus, Ohio, United States
  • The Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
  • Children's Hospital of The King's Daughter Norfolk, Virginia, United States
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Active, not recruiting

International SBMA Project (KDA)

NCT07443449

Conditions
Bulbo-Spinal Atrophy, X-Linked, Central Nervous System Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +11 more
Locations
  • Fondazione IRCCS Istituto Neurologico Carlo Besta Milan, Italy
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Active, not recruiting

Seal, Stopping Eczema and Allergy Study

NCT03742414

Conditions
Atopic Dermatitis, Atopic Dermatitis Eczema, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +13 more
Locations
  • Sean N. Parker Center for Allergy & Asthma Research at Stanford University Palo Alto, California, United States
  • Division of Pediatric Allergy and Clinical Immunology, National Jewish Health Denver, Colorado, United States
  • University of Chicago Chicago, Illinois, United States
  • Cincinnati Children's Hospital Medical Center Cincinnati, Ohio, United States
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