NS-035 / Nippon Shinyaku - LARVOL DELTA

Nippon Shinyaku Co., Ltd. (Headquarters: Kyoto, Japan; President: Toru Nakai) announced that the Ministry of Health, Labor and Welfare (MHLW) has granted Orphan Drug Designation to NS-035 for the treatment of Fukuyama congenital muscular dystrophy (FCMD). (Nippon Shinyaku Press Release) - "NS-035 is a novel antisense oligonucleotide designed to address abnormal protein production caused by mutations in the fukutin gene, the causative gene of FCMD. By promoting the production of functional fukutin protein, NS-035 is expected to suppress disease progression and improve muscle function."

Orphan drug • Muscular Dystrophy