Dissemin is shutting down on January 1st, 2025

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American Association for the Advancement of Science, Science Translational Medicine, 230(6), 2014

DOI: 10.1126/scitranslmed.3008873

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

Journal article published in 2014 by Eric P. Hoffman, Elizabeth M. McNally ORCID
This paper is available in a repository.
This paper is available in a repository.

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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 de-risking and regulatory guidance.