MDA asks Sarepta Therapeutics' CEO Chris Garabedian about next steps for this promising new drug for DMD
MDA is pushing forward on all levels to accelerate the pace at which we’re able to translate scientific discoveries into improved health outcomes for individuals living with muscle disease.
Sarepta Therapeutics answers questions from MDA about its pursuit of regulatory approval for eteplirsen and its development of additional exon-skipping compounds for DMD
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
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.<
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.