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Milo Biotechnology’s FDA Orphan Drug Designation For Muscular Dystrophy

December 28, 2012

Milo Biotechnology’s experimental drug AAV1-FS344 receives 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.


Muscular Dystrophy Association’s (MDA) December 27, 2012 article titled, “MD Briefs: Gene Therapy, Exon Skipping, Stem Cells” December 12, 2012 article/newswire titled, “Milo Biotechnology announces FDA Orphan Drug Designation for AAV1-FS344 for treatment of Duchenne and Becker Muscular Dystrophy”

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