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2 recruiting studies matching your filters
Phase 1
A Phase I/II, Non-Randomized Study of the Safety and Efficacy of In Utero Hematopoietic Stem Cell Transplantation for the Treatment of Fanconi Anemia in Affected Fetuses
The investigators aim to evaluate the safety and efficacy of in utero hematopoietic stem cell transplantation (IUHSCT) for the treatment of fetuses diagnosed w…
Fanconi AnemiaAnemia, Hypoplastic, CongenitalCongenital Bone Marrow Failure Syndromes+5 more
Estimated
~$10 – $8,750
Agnieszka CzechowiczNCT07408583
Phase 3
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy, Safety, and Tolerability of DYNE-251 Administered Intravenously in Ambulatory Male Participants 4 to 18 Years of Age With Duchenne Muscular Dystrophy Amenable to Exon-51 Skipping
The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks…
Duchenne Muscular Dystrophy (DMD)Muscular Dystrophy, DuchenneMuscular Dystrophy (DMD)+10 more
Estimated
~$10 – $8,750
Dyne Therapeutics