AVI BioPharma has begun phase 2 testing of its experimental exon-skipping drug eteplirsen (AVI-4658) in boys with Duchenne MD
Updated: View MDA's video about the launch of the eteplirsen trial on YouTube.
Complete results of a U.K. trial of an exon-skipping drug in boys with DMD show drug is safe, increases dystrophin production
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.
Further analysis of data from a trial of the exon-skipping drug AVI4658 showed some participants had greatly increased dystrophin production
The biopharmaceutical company AVI BioPharma has announced additional encouraging results from its clinical trial of AVI4658, an experimental treatment for Duchenne muscular dystrophy (DMD).
The new results show that, at higher doses, AVI4658 can result in substantial production of the needed dystrophin protein in muscle fibers.
The company has not yet released results of any tests of muscle...