Dyne Therapeutics Inc. (NASDAQ: DYN) receives Orphan Drug designation in Japan for DYNE-251, a therapeutic for Duchenne muscular dystrophy (DMD) with exon 51 skipping mutations. Chief Medical Officer Doug Kerr highlights significant functional improvement and dystrophin expression in ongoing DELIVER trial after 18 months. The drug is part of a global Phase 1/2 trial for rare diseases affecting fewer than 50,000 patients. Dyne focuses on neuromuscular disease therapeutics in the US.

Read more at Yahoo Finance: Dyne’s Investigational DMD Drug DYNE-251 Gets Japan Orphan Drug Status After Strong Trial Data