1) as I noted before SRPT seems to be operating under the assumption that the rules don't apply to them. So the FDA probably has much better evidence of ignoring than usual.
2) what I find sad is that the parents are attacking the wrong group. E.g.:
a) get Sarepta to fix their problems - or help them fix it (if the company is really about the kids they should be happy to let an outside expert, paid for by the parents, come in and help with the assays etc. )
b) move to RNA. Get them to disclose the details of their AE and if monitorable and reversible they should push for RNA, not SRPT (who, almost undeniably, has execution issues generally (even if approved would they be able to manufacture?)). Personally, if I had a child with DMD it would be this tack that I would be taking.
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