exon-skipping

The company says it hopes to have feedback from regulators about its experimental Duchenne MD drug drisapersen by the end of June 2014

posted on April 29, 2014 - 12:49pm
Update (May 1, 2014): For encouraging results from an open-label, extension study of drisapersen (all participants receive the drug in an open-label study), see Prosensa's May 1, 2014, press release.

Complete results of a U.K. trial of an exon-skipping drug in boys with DMD show drug is safe, increases dystrophin production

posted on July 26, 2011 - 11:24am
Complete and extremely encouraging findings from a phase 1b-2 trial of eteplirsen (AVI-4658), an exon skipping drug in development to treat a portion of the Duchenne muscular dystrophy (DMD) population, show the compound is safe and well-tolerated, and that it can significantly increase production of the needed dystrophin protein in recipients without eliciting an unwanted immune response.

A 26-week study by AVI BioPharma finds that intravenous AVI4658 appears safe and beneficial to Duchenne MD patients with mutations in or around exon 51

posted on October 15, 2010 - 1:12pm
The experimental drug AVI4658, in development by AVI BioPharma to treat Duchenne muscular dystrophy (DMD) caused by specific genetic mutations, was well tolerated and resulted in increased production of the needed dystrophin protein. Measured aspects of cardiac, pulmonary and skeletal muscle function remained stable.