Nippon Shinyaku backs EXG-7001 as full-length dystrophin mRNA strategy enters Duchenne race
Discover how Nippon Shinyaku’s EXG-7001 option could advance full-length dystrophin mRNA therapy for Duchenne muscular dystrophy.
Discover how Nippon Shinyaku’s EXG-7001 option could advance full-length dystrophin mRNA therapy for Duchenne muscular dystrophy.
Capricor Therapeutics has secured a July 29 FDA advisory committee meeting for deramiocel after a positive Phase 3 trial revived the previously rejected Duchenne muscular dystrophy application. The therapy now has controlled evidence of skeletal and cardiac benefit, but regulators must still weigh endpoint interpretation, long-term external comparisons, manufacturing reliability and whether the proposed label