You know who you are. pasteur, regulatoryexpert and others.
How can you conduct yourselves in this manner? What drives you to continually suppress your humanity and caring for the poor children with muscular dystrophy?
It is beyond comprehension that somehow your vengeance or financial interests could allow you to shrug your shoulders in favor of denying sick children a chance at survival in deference to proper protocol.
There is no good excuse for your position. I want you to tell everyone here right now how you would feel towards Serepata and Eteplirsen if YOUR child was suffering from this horrific disease.
I've seen outright lies posted here about this treatment.
1) "The FDA loves these kids" (my favorite)
2) "kids were running around" after eteplirsen
3) a new one today "1000 time better than expected"
1) FDA can't have such a close relationship with the patients whose data they are overseeing. Highly improper.
2) Hmmm. isn't it obvious from the 6MWT that it is not the ^M Running T ?
3) I'm not sure of the math behind this claim but
a) the 6MWT results were 1000x better? so I multiply the net decline by 1000x and get a decline that they expected - negative ambulatory distances on the 6MWT? or was it the percentages instead of -0.4% they expected 1000x or a decline of 4x the distance... also yielding negative distances?
b) or was it the dystrophin? one article reported 60% (are they sure they measured right? The FDA wants to know) of normal production. So, divide that by 1000 and you get a nmber so small you would not want to start a trial with an expectation that low.
See where I am going?
This place is not adequately based in reality.
SRPT blew 2 years of money and these kids lives by not even performing proper scientific methods. Now you blame the FDA for asking for the scientific method to be used?
No. Don't blame me, Don't blame the FDA. Blame SRPT.