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Exon Skipping

 

The Exon Skipping COST Action BM1207 aims to advance the development of antisense-mediated exon skipping for rare diseases, focusing on Duchenne muscular dystrophy for which this approach is currently assessed in phase 3 clinical trials.

The project involves all key stake holders (scientists, clinicians, regulators, industry and patients) this COST Action aims to overcome challenges through networking to allow clinical implementation of antisense-mediated exon skipping for as many rare disease patients as possible.

Exon Skipping
Project started April 2013 and will end April 2017

Funded by COST

Coordinated by

Annemieke Aartsma-Rus - Leiden University Medical Center, Netherlands




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