Clinical study on gene therapy for Duchenne muscular dystrophy (MDM)
NS-065 / NCNP-01 is an exon-skipping nucleic acid drug created through joint research between National Center for Neurological and Medical Research (NCNP) and Nippon Shinyaku. Itt is targeted as a remedy for patients with DMD who have gene mutations responding to exons 53 skipping of the dystrophin gene. In this study, NS – 065 / […]