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

Iff J, Done N, Tuttle E, Zhong Y, Wei F, Darras BT, McDonald CM, Mercuri E, Muntoni F
Muscle Nerve. 2024 Jul;70(1):60-70. doi: 10.1002/mus.28075. Epub 2024 Mar 14. PMID: 38482981
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.
Not Epub
Organism or Cell Type: 
Delivery Method: 
i.v. infusion