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.

Reports from the American Academy of Neurology annual meeting about Duchenne, limb-girdle and myotonic muscular dystrophies, dermatomyositis, myasthenia gravis and spinal muscular atrophy

posted on April 20, 2011 - 4:49pm
Below are brief reports and links to more information about neuromuscular disease research presented at the 63rd annual meeting of the American Academy of Neurology (AAN), held in Honolulu April 9-16, 2011.

Further analysis of data from a trial of the exon-skipping drug AVI4658 showed some participants had greatly increased dystrophin production

posted on June 2, 2010 - 10:15am
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...
posted on January 1, 2008 - 11:16am
QUEST Vol. 15, No. 1
This article reports on clinical trials in: Duchenne muscular dystrophy, Friedreich's ataxia and myotonic dystrophy.