Sarepta Therapeutics answers questions from MDA about its pursuit of regulatory approval for eteplirsen and its development of additional exon-skipping compounds for DMD
posted on December 7, 2012 - 4:55pm
In both October and December 2012, Sarepta Therapeutics announced very encouraging results from a 12-person phase 2b trial of eteplirsen, an exon-skipping compound that is a potential treatment for Duchenne muscular dystrophy.
Eteplirsen targets exon 51 of the dystrophin gene and is designed to treat DMD caused by specific dystrophin mutations.
Sarepta Therapeutics plans to target three more dystrophin exons, in addition to exon 51, as potential treatments for Duchenne MD
posted on December 4, 2012 - 10:45am
Biopharmaceutical company Sarepta Therapeutics has announced it will expand the focus of its exon-skipping program for Duchenne muscular dystrophy (DMD) by developing compounds that target exons 45, 50 and 53 of the dystrophin gene, in addition to continuing to develop eteplirsen, which targets exon 51 of this gene.<