Exon-skipping therapy: A roadblock, detour, or bump in the road?

Eric P. Hoffman*, Elizabeth M. McNally

*Corresponding author for this work

Research output: Contribution to journalShort surveypeer-review

28 Scopus citations


Exon skipping is a promising therapeutic for Duchenne muscular dystrophy patients, but the road to drug approvals is foggy and may require more early-stage derisking and regulatory guidance.

Original languageEnglish (US)
Article number230fs14
JournalScience translational medicine
Issue number230
StatePublished - Apr 2 2014

ASJC Scopus subject areas

  • Medicine(all)


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