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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The author would like to thank Kyla Dunn for critical reading and editing.
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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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DOI: https://doi.org/10.1038/nrneurol.2016.180
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