The American Academy of Neurology's Guidelines Subcommittee has reviewed the efficacy and adverse effects of delandistrogene moxeparvovec in Duchenne muscular dystrophy patients, highlighting limitations in clinical benefit despite FDA approval. Studies showed minimal improvements in secondary motor outcomes, raised concerns about safety, and identified limitations in using expression of microdystrophin as a surrogate endpoint. Providers are advised to monitor for immune-related side effects, acknowledging the treatment's uncertainties and the need for further evidence to establish its effectiveness and safety across ages.

Source: Neurology