“Duchenne muscular dystrophy has seen more progress in the past 15 months (give or take) than in the last couple of decades combined. In that time, the Food and Drug Administration has approved three new therapies: Elevidys (delandistrogene moxeparvovec-rokl), the first micro-dystrophin gene therapy; Duvyzat (givinostat), a non-steroidal HDAC inhibitor; and Agamree (vamorolone), a new corticosteroid.”

From STAT.