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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.

12,656 public trials 2,147 recruiting studies
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Genetic Diseases, Inborn trial results

Completed

Treatment of Graves' Orbitopathy (Thyroid Eye Disease) to Reduce Proptosis With Teprotumumab Infusions in a Randomized, Placebo-Controlled, Clinical Study

NCT03298867

Conditions
Autoimmune Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Endocrine System Diseases, +13 more
Locations
  • Macro, Llc Beverly Hills, California, United States
  • Cedars-Sinai Medical Center Los Angeles, California, United States
  • The Lennar Foundation Medical Coral Gables, Florida, United States
  • Bascom Palmer Eye Institute Miami, Florida, United States
View all 14 locations
View Trial Details
Completed

Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)

NCT01347073

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +13 more
Locations
  • UCLA Pediatrics/Genetics Los Angeles, California, United States
  • Children's National Medical Center Washington D.C., District of Columbia, United States
  • Maine Medical Center Portland, Maine, United States
  • University of Minnesota Minneapolis, Minnesota, United States
View Trial Details
Completed

Study of the Safety of HPN (Hyperion)-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD)

NCT00947297

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +13 more
Locations
  • Long Beach Memorial Long Beach, California, United States
  • UCLA Los Angeles, California, United States
  • Stanford University Stanford, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
View all 22 locations
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Completed

Hydroxyurea to Prevent Brain Injury in Sickle Cell Disease

NCT01389024

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +14 more
Locations
  • University of Alabama Birmingham, Alabama, United States
  • Johns Hopkins Hospital Baltimore, Maryland, United States
  • Mercy Children's Hospital Kansas City, Missouri, United States
  • St. Louis Children's Hospital St Louis, Missouri, United States
View Trial Details
Completed

Efficacy and Safety of Losmapimod in Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD)

NCT04003974

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Facioscapulohumeral Muscular Dystrophy (FSHD), Genetic Diseases, Inborn, +7 more
Locations
  • University of California Irvine Irvine, California, United States
  • University of California Los Angeles (UCLA) Los Angeles, California, United States
  • University of Florida Gainesville, Florida, United States
  • University of Kansas Medical Center Kansas City, Kansas, United States
View all 17 locations
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Completed

Study to Assess the Safety, Pharmacokinetics and Efficacy of KRN23 in Adult Chinese Patients With XLH

NCT04842019

Conditions
Avitaminosis, Bone Diseases, Bone Diseases, Metabolic, +26 more
Locations
  • Peking Union Medical College Hospital Beijing, China
  • The First Medical Center of Chinese People's Liberation Army General Hospital Beijing, China
  • Nanfang Hospital Guangzhou, China
  • The Second Affiliated Hospital of Zhejiang University Hangzhou, China
View Trial Details
Completed

Efficacy and Safety of HPN-100 for the Treatment of Adults With Urea Cycle Disorders

NCT00992459

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +13 more
Locations
  • Long Beach Memorial Long Beach, California, United States
  • UCLA Los Angeles, California, United States
  • Stanford University Stanford, California, United States
  • Denver Children's Hospital Aurora, Colorado, United States
View all 22 locations
View Trial Details

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