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MDA Collaborates with AVI BioPharma on First Phase 2 Placebo-Controlled Clinical Trial of Exon-51 Skipping Drug as Potential Therapy for Duchenne Muscular Dystrophy
Date:8/15/2011

Ohio State University. "Today, we're underway with a clinical trial of a drug that ultimately could create a shortened but functional dystrophin protein for DMD boys with certain out-of-frame gene deletions that may be corrected by skipping exon 51."

"By administering eteplirsen by i.v. for 24 weeks," Mendell added, "our goal is to find the best dosage to trick the body into skipping over genetic disruptions present in some cases of DMD, to produce dystrophin levels typical of Becker muscular dystrophy.  In Becker many patients are able to walk into late adulthood and to have normal or near normal life spans."

The introduction of exon skipping to restore the open reading frame using splice-switching oligomers targeting dystrophin exons is one of several attractive therapeutic strategies for Duchenne muscular dystrophy being pioneered by MDA-funded investigators.  

"MDA has been funding exon skipping research for Duchenne muscular dystrophy for more than a decade," noted Valerie Cwik, M.D., MDA Executive Vice President – Research and Medical Director.  "We're very pleased to now be collaborating with AVI BioPharma on this trial and are hopeful that eteplirsen will become an effective therapy for those living with Duchenne muscular dystrophy."

About MDA

MDA is the nonprofit health agency dedicated to curing muscular dystrophy, ALS and related diseases by funding worldwide research.  The Association also provides comprehensive healthcare and support services, advocacy and education.  

In addition to funding more than 300 research teams worldwide, MDA maintains a national network of some 200 hospital-affiliated clinics; orchestrates hundreds of support groups for families affected by neuromuscular diseases; and facilitates extraordinary local summer camp opportunities for thousands of youngsters fighting progressive muscle diseases.  The Association i
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SOURCE Muscular Dystrophy Association
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