"They sill die prematurely, but not as prematurely. "
and the matched age analysis showed that the Etep patients declined at the same rate as the "control". it's right there in the graphs. hard to argue with hard facts, but if you're goal is to extract $X00,000/boy/year such details don't matter.
nice sound bite, but Etep doesn't, even in SRPT's most propagandist moments, cure DMD. at best, it extends mobility for a little while. nothing more.
you obviously didn't read the briefer. FDA repeated that it had told SRPT to design a proper trial numerous times, which SRPT idnored.
sure. and they'll burn that cash on a PIII, despite the PII results. they'll data-mine to find some subset and go off, again, to find the holy grail. wouldn't be the first time in bio that bad PII led to PIII, you know.
reaction, perhaps, to the "60 Minutes" piece on using polio (degraded) virus directly on brain cancers. using virus/bacteria as carrier may be supplanted by using them directly as therapy????
the PR made ambiguous reference to something not delivered. didn't say what, and they didn't say on the CC. remember: one way out of chap. 11 is to cancel outstanding shares and re-capitalize. 11 doesn't mean current shareholders won't get killed as they are in 7. 11 come 7 is craps backwards.
-- There is no connection in any way between this drug and thalidomide
there sure is: thalidomide was "approved" in Europe on the basis of shoddy review. doctors were free to prescribe in any way they saw fit. if FDA abdicates its responsibility over approval, doctors will prescribe all sorts of snake oil and more thalidomides will happen. I guaronteee it.
-- the decision to use Sarepta should be a patient /doctor one
So, you want another thalidomide disaster? which we didn't have here just because FDA had the balls to say NO.
"(ii) delivery to PharmAthene of 100% of newly-issued stock of SIGA, with all existing shares of SIGA's common stock being cancelled with no distribution to existing shareholders on account thereof"
-- and dilute the hell out of the rest of us? then reverse?
why bother? the report says that PIP can be given 100% of the company and current shares cancelled.
for SNTA? reads like Mad. is taking SNTA's cash to fund Mad.'s pipeline. why is this good for SNTA, which has/had late stage assets?
superficially, at least, it looks like Madrigal is the beauty and Synta the beast. in fact, one wonders what Madrigal wants from Synta? they ain't of the Biotech Beltway, either.
one expects smaller delta from PII to PIII, but here we have actual reversal of effect. that's going to be difficult to explain, without using the "f" word. ok, out of the gutter. the word is "fraud".
low on $$$ will sell shares very soon. those with dry powder waiting. it isn't as if cash flow positive is right around the corner.
the larger the delta, the smaller the sample to reach a specified p-value (if you still think p-value matters, i.e. your not a Bayesian).