8 Emerging Therapies Shaping the Muscular Dystrophy Treatment Landscape

Emerging therapies in muscular dystrophy include RNA-based treatments, cell therapy, and molecular approaches targeting disease mechanisms like exon skipping and DUX4 suppression. Notable regulatory developments include Dyne Therapeutics submitting a biologics license application for z-rostudirsen and FDA acceptance of Capricor Therapeutics' application for deramiocel, a potential first cell therapy for Duchenne muscular dystrophy cardiomyopathy.

This Wire brief sits within Fusion42's coverage of Biotech.