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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.

456 public trials 107 recruiting studies
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Genetic Diseases, Inborn trial results

Recruiting

Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia

NCT07441876

Conditions
Achondroplasia, Bone Diseases, Bone Diseases, Developmental, +5 more
Locations
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • Nemours Children's Health Wilmington, Delaware, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • Johns Hopkins Medicine Baltimore, Maryland, United States
View all 28 locations
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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

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
View all 15 locations
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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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