You are here

Survival among patients receiving eteplirsen for up to 8 years for the treatment of Duchenne muscular dystrophy and contextualization with natural history controls

Authors: 
Iff J, Done N, Tuttle E, Zhong Y, Wei F, Darras BT, McDonald CM, Mercuri E, Muntoni F
Citation: 
Muscle Nerve. 2024 Jul;70(1):60-70. doi: 10.1002/mus.28075. Epub 2024 Mar 14. PMID: 38482981
Abstract: 
Eteplirsen, approved in the US for patients with Duchenne muscular dystrophy (DMD) with exon 51 skip-amenable variants, is associated with attenuated ambulatory/pulmonary decline versus DMD natural history (NH). We report overall survival in a US cohort receiving eteplirsen and contextualize these outcomes versus DMD NH.
Epub: 
Not Epub
Link to Publication: 
https://onlinelibrary.wiley.com/doi/10.1002/mus.28075
Organism or Cell Type: 
human
Delivery Method: 
i.v. infusion