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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.

453 public trials 108 recruiting studies
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Genetic Diseases, Inborn trial results

Recruiting

A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping

NCT07587242

Conditions
Congenital, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DMD, +22 more
Locations
  • AP-HP Hospital Armand-Trousseau Paris, France
  • Universitaetsklinikum Essen Essen, Germany
  • Universitaetsklinikum Heidelberg Heidelberg, Germany
  • Klinikum der Ludwig-Maximilians-Universitaet Muenchen München, Germany
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Recruiting

Screening Protocol for Genetic Diseases of Lymphocyte Homeostasis and Programmed Cell Death

NCT00246857

Conditions
Autoimmune Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +4 more
Locations
  • National Institutes of Health Clinical Center Bethesda, Maryland, United States
  • University of Michigan Ann Arbor, Michigan, United States
  • Ankara Medical University Ankara, Turkey (Türkiye)
  • Gazi University Ankara, Turkey (Türkiye)
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Recruiting

Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study

NCT06016946

Conditions
Ataxia, Brain Diseases, Central Nervous System Diseases, +19 more
Locations
  • UCLA Ataxia Center Los Angeles, California, United States
  • University of Colorado Denver, Colorado, United States
  • University of Florida - Neurology Gainesville, Florida, United States
  • USF Ataxia Research Center Tampa, Florida, United States
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Recruiting

Auditory Restoration and Outcomes After Gene Therapy and Cochlear Implantation: a PRospective Cohort Analysis (AURORA)

NCT06237790

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Ear Diseases, Genetic Disease, +10 more
Locations
  • Peking Union Medical College Hospital Beijing, Beijing Municipality, China
  • The First Affiliated Hospital of Zhengzhou University Zhengzhou, Henan, China
  • The Second Xiangya Hospital of Central South University Changsha, Hunan, China
  • The First Affiliated Hospital of Nanchang University Nanchang, Jiangxi, China
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Recruiting

Familial Mediterranean Fever and Related Disorders: Genetics and Disease Characteristics

NCT00001373

Conditions
ALPK1, Autoinflammation, Body Temperature Changes, +26 more
Locations
  • Childrens National Medical Center Washington D.C., District of Columbia, United States
  • Johns Hopkins University Baltimore, Maryland, United States
  • Walter Reed National Medical Center Bethesda, Maryland, United States
  • National Institutes of Health Clinical Center Bethesda, Maryland, United States
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Recruiting

Clinical and Genetic Evaluation of Individuals With Undiagnosed Disorders Through the Undiagnosed Diseases Network

NCT02450851

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Genetic Disease, +5 more
Locations
  • University of Alabama at Birmingham Birmingham, Alabama, United States
  • HudsonAlpha Institute for Biotechnology, Inc. Huntsville, Alabama, United States
  • University of California, Los Angeles Los Angeles, California, United States
  • University of California, Irvine Medical Center Orange, California, United States
View all 33 locations
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