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A mini review and implementation model for using ataluren to treat nonsense mutation Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD) is a fatal neuromuscular disease caused by mutations in the dystrophin gene. Approximately 10-15% of cases result from nonsense mutations (nmDMD), which ataluren targets to restore functional dystrophin. This review provides clinical guidelines for ataluren use and proposes an implementation model in Sweden. The study conducted a targeted review of literature (1995-2018), including trials, guidelines, and commentaries on nmDMD and ataluren. It focused on treatment …
