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A New Orphan Drug For Duchenne Muscular Dystrophy ?

December 12, 2012

       Milo Biotechnology’s experimental drug AAV1-FS344 is given FDA Orphan Drug Designation (ODD) on November 19, 2012, for the treatment of Duchenne and Becker Muscular Dystrophy (DMD & BMD, respectively). Orphan drug AAV1-FS344 is a “gene delivery therapy that produces Follistatin, a protein that increases muscle mass and strength”, in patients with both types of Muscular Dystrophy. 

        A Phase I clinical trial, funded by the Parent Project Muscular Dystrophy (PPMD) foundation, is underway for AAV1-FS344 in adults with BMD and inclusion body myositis, at the Nationwide Children’s Hospital in Columbus, Ohio.

References 

MedCityNews.com December 12, 2012 article titled, “New follistatin gene therapy for muscular dystrophy gets orphan drug status”   

StreetInsider.com December 12, 2012 article/newswire titled, “Milo Biotechnology announces FDA Orphan Drug Designation for AAV1-FS344 for treatment of Duchenne and Becker Muscular Dystrophy”  

The Myositis Association

 Copyright © 2012-2013, Orphan Druganaut Blog.  All rights reserved.

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