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.

Exonskipping logo
Project start date – April 2013
Project end date – April 2017

Funded by COST

Coordinated by

Annemieke Aartsma-Rus - Leiden University Medical Center, Netherlands