Trial results for the Duchenne MD drug eteplirsen show real improvements in dystrophin production and walking ability
Update (Aug. 8, 2013): This story has been updated to reflect the Aug. 1, 2013, publication of these results in Annals of Neurology.
A trial of the exon-skipping drug eteplirsen resulted in an increase in both dystrophin production and walking ability in boys with Duchenne MD
Update (Aug. 8, 2013): This story has been updated to reflect the Aug. 1, 2013, publication of these trial results in Annals of Neurology.
The developer of a promising exon-skipping drug for Duchenne MD explains its clinical trial program and current limits on access to the drug
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.
Boys with Duchenne MD who received 36 weekly infusions of an experimental exon-skipping drug showed a significantly slower decline in walking ability
Editor's note (July 30, 2012): This story was revised to include information about the specific mutations being targeted by eteplirsen.