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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.

12,578 public trials 2,158 recruiting studies
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Genetic Diseases, Inborn trial results

Enrolling by Invitation

An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2 Who Have Completed MEX-DM-302 Study.

NCT06549400

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, +9 more
Locations
  • Laboratory for Muscle Diseases and Neuropathies Leuven, Belgium
  • Aarhus University Hospital Aarhus, Denmark
  • Ludug-Maximilians University München, Germany
  • Azienda Ospedaliera Universitaria Policlinico Tor Vergata Rome, Italy
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Enrolling by Invitation

An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects

NCT05594992

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +16 more
Locations
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
  • Hospital Universitario Austral Buenos Aires, Argentina
  • Hospital de Clínicas de Porto Alegre Porto Alegre, Brazil
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Enrolling by Invitation

Alpha-1 Foundation DNA and Tissue Bank

NCT00884455

Conditions
Alpha 1 Antitrypsin Deficiency, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, +9 more
Locations
  • University of Florida Gainesville, Florida, United States
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