exon skipping drug

Drisapersen, designed to treat boys with Duchenne MD with mutations near exon 51 of the dystrophin gene, showed no benefit in a phase 3 trial

posted on September 20, 2013 - 12:55pm
Update (Dec. 19, 2013): In a communication today to patient groups, GlaxoSmithKline (GSK) said that, together with Prosensa, it is continuing to analyze the phase 3 drisapersen trial and other aspects of the drisapersen clinical program, and that it anticipates having results of this analysis in January 2014. Until then, GSK said, dosing of drisapersen in ongoing studies will continue to be...

The developer of a promising exon-skipping drug for Duchenne MD explains its clinical trial program and current limits on access to the drug

posted on August 31, 2012 - 2:55pm
In July 2012, Sarepta Therapeutics announced encouraging interim results for its phase 2b trial of eteplirsen, an experimental exon-skipping drug in development to treat Duchenne muscular dystrophy (DMD) caused by specific mutations in the dystrophin gene.