Browse Studies — Arctuva

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

Observational

North American Fetal Therapy Network Prospective Registry for Long-term Outcome Following Fetoscopic Endoluminal Tracheal Occlusion in Severe Left and Right Congenital Diaphragmatic Hernia

This registry study aims to confirm that FETO increases neonatal survival to discharge and reduces long-term morbidity in fetuses with isolated left CDH and o/…

Congenital Diaphragmatic HerniaCongenital AbnormalitiesNeonatal Diseases and Abnormalities+5 more

Estimated

~$10 – $6,250

Johns Hopkins UniversityNCT07166172