In late 2012, we awarded a small grant to be involved in the SKIP-NMD programme.  SKIP-NMD is an EU FP7 funded collaborative grant involving 10 partners from Europe and the USA, whose aim is to restore dystrophin production in a subset of DMD boys. This will be achieved by developing a drug which ‘skips’ exon 53 the mutations causing DMD, so as to restore dystrophin protein expression.

This exon skipping compound is based on Sarepta Therapeutic’s PMO AO chemistry and will potentially treat boys with deletions spanning exons 52, 45-52, 47-52, 48-52, 49-52 and 50-52 (about 6% of the population). The drug will first undergo pre-clinical tests, followed by a phase I/IIa clinical trial. The project will also develop new outcome measures and biomarkers to ascertain the drug’s effectiveness.