WATCH TIME: 3 minutes "One possibility is that prescribing physicians arent aware of the age range that received the drug in its pivotal trials or have knowledge on the FDA-approval process. When patients receive the drug at a later stage of their DMD, it might impact the predicted effectiveness of the medications." For years, Duchenne muscular dystrophy (DMD) was mainly treated with a combination of corticosteroids, assistive devices, and cardiac and respiratory support until 2016, when the exon-skipping therapies began to emerge. The first approved therapy was eteplirsen (Exondys 51; Sarepta Therapeutics), an antisense oligonucleotide designed to increase dystrophin, followed by other agents, which include golodirsen, viltolarsen, and casimersen. Continue reading →