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Friday, April 26, 2013

DMD Expert

While there seems to be a debate related to the dystrophin being produced by eteplirsen and whether or not it has proven to be correlated with a clinical benefit, I believe the FDA guidance on accelerated approval requires dystrophin production only be "reasonably likely to predict" clinical benefit.

http://www.thestreet.com/story/11906821/1/a-dmd-expert-and-mother-calls-for-eteplirsens-rapid-approval.html

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