The New England Journal of Medicine Publishes Results from Phase 1/2 MARINA® Trial of Delpacibart Etedesiran (del-desiran) for Treatment of Myotonic Dystrophy Type 1

Del-desiran effectively delivered siRNA to muscle, resulting in approximately 40% mean reduction in DMPK mRNA and amelioration of missplicing Treatment demonstrated improvements in multiple measures including myotonia, muscle function and strength, mobility and patient-reported outcomes...