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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.

12,573 public trials 2,159 recruiting studies
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Genetic Diseases, Inborn trial results

Enrolling by Invitation

Global Open-Label Extension Study of Del-desiran for the Treatment of DM1

NCT07008469

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DM1, Genetic Diseases, Inborn, +19 more
Locations
  • University of California, Los Angeles (UCLA) Los Angeles, California, United States
  • Stanford University Stanford, California, United States
  • University of Colorado Denver, Colorado, United States
  • University of Florida Gainesville, Florida, United States
View all 35 locations
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Enrolling by Invitation

AI Echocardiographic Screening of Cardiac Amyloidosis

NCT06664866

Conditions
Amyloid Neuropathies, Amyloid Neuropathies, Familial, Amyloidosis, +13 more
Locations
  • Cedars Sinai Medical Center Los Angeles, California, United States
  • Palo Alto Veteran Affairs Hospital Palo Alto, California, United States
  • Northwestern Medicine Chicago, Illinois, United States
  • Providence Heart and Vascular Institute Portland, Oregon, United States
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Enrolling by Invitation

Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema

NCT05396105

Conditions
Angioedema, Angioedemas, Hereditary, C1 Esterase Inhibitor Deficiency, +30 more
Locations
  • Study site Birmingham, Alabama, United States
  • Study site Little Rock, Arkansas, United States
  • Study site San Diego, California, United States
  • Study site Santa Monica, California, United States
View all 60 locations
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Enrolling by Invitation

Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA

NCT04360265

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +13 more
Locations
  • Nationwide Children's Hospital Columbus, Ohio, United States
  • Women's and Children's Hospital North Adelaide, South Australia, Australia
  • Vall d'Hebron Barcelona Campus Barcelona, Spain
  • Hospital Clínico Universitario de Santiago Santiago de Compostela, Spain
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Enrolling by Invitation

Phase 1b Long-term Extension Trial of RAY121 in Immunological Diseases (RAINBOW-LTE Trial)

NCT06723106

Conditions
Antiphospholipid Syndrome, Antiphospholipid Syndrome (APS), Autoimmune Diseases, +50 more
Locations
  • Royal Prince Alfred Hospital Camperdown, New South Wales, Australia
  • Westmead Hospital Sydney, New South Wales, Australia
  • Campbelltown Public Hospital Sydney, New South Wales, Australia
  • The Alfred Hospital Melbourne, Victoria, Australia
View all 62 locations
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