Duchenne muscular dystrophy (DMD) is a muscle degeneration disorder caused by mutations affecting the dystrophin gene. Researchers show how a dual CRISPR RNA method restored dystrophin protein ...

Genentech announced positive data from the pivotal part 2 of the SUNFISH trial that evaluated the safety and efficacy of risdiplam in adult and pediatric patients with Type 2 or Type 3 spinal muscular ...