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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 Muntoni
Published 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.
Keyphrases
  • duchenne muscular dystrophy
  • muscular dystrophy
  • free survival
  • electronic health record
  • big data
  • machine learning
  • combination therapy