Clinical Research Directory
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2 clinical studies listed.
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Tundra lists 2 Congenital, Hereditary, and Neonatal Diseases and Abnormalities clinical trials. Each listing includes eligibility criteria, study locations, and direct links to research sites in the Tundra directory.
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NCT07608432
Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)
The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks to ambulatory Duchenne muscular dystrophy (DMD) participants, 4 to 18 years of age, with dystrophin mutations amenable to exon 51 skipping.
Gender: MALE
Ages: 4 Years - 18 Years
Updated: 2026-05-27
1 state
NCT07408583
Prenatal Transplantation for Fetuses With Fanconi Anemia
The investigators aim to evaluate the safety and efficacy of in utero hematopoietic stem cell transplantation (IUHSCT) for the treatment of fetuses diagnosed with Fanconi anemia (FA) during pregnancy.
Gender: All
Updated: 2026-02-19
1 state