PER 0.00% 7.2¢ percheron therapeutics limited

Thanks @Uboy,So, lets go through this again to check that we...

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    Thanks @Uboy,
    So, lets go through this again to check that we have the gist of the argument for the accelerated approval of SRP-9001. The post hoc analysis by Sarepta demonstrated that 4-5 year olds showed improvement compared to placebo while 6-7 year olds did worse than placebo. Its unsurprising that the FDA might provide for restricted approval for the younger cohort. But there is something missing from the analysis, there is an elephant in the room that apparently Sarepta doesn't appear to address? Why does turning six suck!!!

    From my reading to date I haven't seen anybody else ask the question, neither the FDA nor advocacy groups. Sarepta might have a vested interest in keeping mum but surely the medical profession must have a clue. Of course they do and as you say the scientific literature points to a lot of evidence that fibrosis plays a big role in the decline of DMD boys after the age of five.

    Time may be muscle but time also turns to fat due to the vicious cycle of inflammation and fibrosis. I think the FDA will eventually connect the dots.
 
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