Parent advocacy of molecular medicine for Duchenne muscular dystrophy

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Jon Moulton
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Parent advocacy of molecular medicine for Duchenne muscular dystrophy

Here are a few parent-advocacy articles on Duchenne muscular dystrophy and eteplirsen, a Morpholino antisense drug in clinical trials.  Eteplirsen causes skipping of exon 51 in the dystrophin pre-mRNA, causing the reading frame to shift downtream of the skip.  It is used to correct the reading frame in those with DMD that have frameshift mutations very near exon 51.
http://www.huffingtonpost.com/tracy-seckler/eteplirsen-duchenne_b_4904850.html
http://www.huffingtonpost.com/lauren-caruba/a-race-against-the-clock-_b_4900631.html