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Eteplirsen therapy for Duchenne muscular dystrophy: skipping to the front of the line

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New therapies are much needed for Duchenne muscular dystrophy. Recent data from a phase II clinical trial has led to accelerated FDA approval of the exon-skipping drug eteplirsen. This approval is provisional, pending results of an ongoing phase III clinical trial, and came after much debate.

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Acknowledgements

The author would like to thank Kyla Dunn for critical reading and editing.

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  1. Division of Neurology and Program for Genetics and Genome Biology, Hospital for Sick Children, 555 University Avenue, Toronto, M5G 1X8, Ontario, Canada

    James J. Dowling

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  1. James J. Dowling
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Correspondence to James J. Dowling.

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Dowling, J. Eteplirsen therapy for Duchenne muscular dystrophy: skipping to the front of the line. Nat Rev Neurol 12, 675–676 (2016). https://doi.org/10.1038/nrneurol.2016.180

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