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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

A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

NCT07354724

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • University of California-Irvine Irvine, California, United States
  • Duke University School of Medicine - Early Phase Research Unit Durham, North Carolina, United States
  • The Lysosomal & Rare Disorders Research & Treatment Center Fairfax, Virginia, United States
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Recruiting

Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant

NCT06563895

Conditions
Alzheimer Disease, Amyloid Cardiomyopathy, Amyloid Neuropathies, +26 more
Locations
  • University of California, San Diego (UCSD) - Medical Center La Jolla, California, United States
  • University of California, Los Angeles (UCLA) - David Geffen School of Medicine Los Angeles, California, United States
  • University of California, San Francisco (UCSF) San Francisco, California, United States
  • Stanford University Stanford, California, United States
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Recruiting

Integrated Cancer Repository for Cancer Research

NCT02012699

Conditions
Abdominal Neoplasms, Adenoma, Adenomatous Polyposis Coli, +195 more
Locations
  • Advent Health Greenwood Village, Colorado, United States
  • Hartford HealthCare Cancer Institute at Manchester Medical Hospital Manchester, Connecticut, United States
  • Florida Hospital Memorial Medical Center Daytona Beach, Florida, United States
  • Florida Hospital DeLand DeLand, Florida, United States
View all 42 locations
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Recruiting

A Study to Evaluate the Tolerability, Safety and Efficacy of GNR-097 Gene Therapy in Pediatric Patients With Duchenne Muscular Dystrophy

NCT07673809

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations
  • Republican Scientific and Practical Center Mother and Child Minsk, Belarus
  • Russian Children's Clinical Hospital Moscow, Russia
  • National Medical Research Center for Children Moscow, Russia
  • Veltischev Research and Clinical Institute for Pediatrics and Pediatric Surgery of the Pirogov Russian National Research Medical University Moscow, Russia
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Recruiting

An Extension Study to Evaluate Safety and Efficacy of Atumelnant in Participants With Congenital Adrenal Hyperplasia

NCT06712823

Conditions
Adrenal Gland Diseases, Adrenal Hyperplasia, Congenital, Adrenogenital Syndrome, +17 more
Locations
  • Crinetics Study Site, Minneapolis, Minnesota 55454 Minneapolis, Minnesota, United States
  • Crinetics Study Site Morehead City, North Carolina, United States
  • Crinetics Study Site Philadelphia, Pennsylvania, United States
  • Crinetics Study Site Córdoba, Córdoba Province, Argentina
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