Two things - The FDA isn't "urgent" - they are a regulatory agency whose charter is to PREVENT dangerous and/or ineffective treatments from reaching the market; that they have recognized that there are situations where the long regulatory lead times are unduly burdensome for certain treatments and therapies is a positive, but we should never forget that their principal focus is the prevention of bad medicine not the facilitation of good medicine. Second: NOBODY "pushes" the FDA. In fact the field of biotech is littered with the dead and wounded who either tried to "push" them or were adequately aggressive in their public statements to give the appearance of even TRYING to "push" them.
Jenn and the rest of the DMD community have done an exceptional job of putting a face to this horrid disease and a variety of FDA officials have expressed their appreciation for that input. That's a big positive. Sarepta has done an excellent job in responding to FDA requests for information and in keeping shareholders informed as to progress WITHOUT giving the appearance of "pressuring the Agency. As a matter of fact I don't believe I've ever seen organizations do a better job of being informative and reasonably transparent WITHOUT crossing the line into territory that might be viewed as being inappropriate by the Agency.
Now it's time to be patient. The drug is effective and has no material safety issues. The options for the patient population at this point are this treatment or decline and death. Both of these points have been amply made. The DMD parents and Sarepta have done their job - now we need to let the FDA do theirs.
I don't know how much harder this lady and the other parent advocates can push. They seem to be doing everything in their power. The people on this board are here primarily for selfish reasons and that is to hopefully make money through stock appreciation. The parents on the other hand, are guided by love and affection for their children, and if you are a parent you know precisely what I am talking about, so in the long run we all have a common goal in wanting SRPT to be successful.
I think the biggest issue remaining to be resolved is CMC, with emphasis on "M". My gut tells me the FDA needs assurances on this. And if you don't know what I am talking about, look it up.