FocusDuchenne Muscular Dystrophy

Exon-Skipping Therapy: A Roadblock, Detour, or Bump in the Road?

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Science Translational Medicine  02 Apr 2014:
Vol. 6, Issue 230, pp. 230fs14
DOI: 10.1126/scitranslmed.3008873

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Abstract

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.

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