Survival among patients receiving eteplirsen for up to 8 years for the treatment of Duchenne muscular dystrophy and contextualization with natural history controls.
Joel IffNicolae DoneEdward TuttleYi ZhongFangzhou WeiBasil T DarrasCraig M McDonaldEugenio MercuriFrancesco MuntoniPublished in: Muscle & nerve (2024)
Data suggest eteplirsen may prolong survival in patients with DMD across a wide age range. As more data become available, the impact of eteplirsen on survival will be further elucidated.