nothing saves lives, only prolongs them. plenty of evidence of that.
On average the nationwide number of potential thoracic enrollees is 367/month. Now exclude those not complete, not meeting other admission criteria, seasonality, regionality in site areas, lack of familiarity in hospitals and trauma centers, for whatever reason not willing to transfer a surgical patient from an admission hospital to a study hospital, those not willing to to be part of a study and the price of tulips in Denmark and your pool dwindles quickly. They're doing just fine. Expect another within a month.
not sure what Johnny Walker has to do with this but when you find out in ignoreland send a message.
interesting thought, that 10% is needed. I haven't been able to find any data from studies showing any clinical correlation between threshold amounts of dystrophin and functional endpoints. In fact the only inference I've seen is from the Eteplirsen data. Can you reference your statement? TIA
God knows I hate the expression watch and learn but perhaps this is the time when it is in everyone's best interest to do just that.
from FDA BD for pimavanserin for the treatment of psychosis in Parkinson’s disease. Interesting thing is it was written as a lead in to reasons this drug was unsafe.
quote from the FDA's Dr. Mitchell Mathis (oversees the FDA's division of psychiatry products):
if a “… drug is completely without safety signals, then we need not spend too much time defining clinical meaningfulness because benefit, however small, outweighs zero risk, and even a small benefit in a disabling disease is valuable.”
and were they approved by the FDA senior staff?
did they post any of the speaker slides that they were mysteriously unable to show during pubic comment?
and I'll be here to graciously admit I was wrong. And happily I might add. And I promise not to gloat if I'm correct or closer to what is than your PT.
well I admit I've been wrong before, which is why I'm more careful with valuations vs market reaction to events. Hopefully I'm wrong again but I'll stick with mine for the time being and we'll revisit this soon I hope.
Guys like me? Just who are guys like me and whom am I being lumped into a category with?
but I do. shorts and their machines know how to control the price so it doesn't get out of hand for them. They hedge with option spreads and and many of them are already playing both ends. I don't know current status but in June last year when the filed the NDA they made an agreement with Midcap Financial to secure $40 million in debt financing. The debt matures in four years and bears a pretty high annual interest rate of 7.75%. Sarepta noted that it had already drawn down $20 million against its new term loan.
your mouth to God's ears as they say. However, that is an ambitious evaluation depending on actual pricing, insurance coverage, and stipulations concerning forward data gathering and other factors, some of which may be face saving. I think you see a jump to $35-$40 with FDA approval followed by some give back. The real money will comes with a BP partner ( I still see Pfizer as a suitor) or buy out. Strategy to consider, sell half or more on the announcement or soon after and re accumulate after the give back. European approval adds value. solid data from the next phase studies will take it to $100 or more if it hasn't been taken out. GL to the families and to faithful longs.