I really hope, if nothing else, that Austin qualifies to be accepted into the non ambulatory arm of the upcoming trial. Lets watch as Eteplirsen rescues his ability to play those drums! Now THAT is what I'm talking about, and I hope it happens.
Greyzone bagging is not allowed with FDA as they (FDA) cannot play with rest of the people's life. Pharma business is in Trillions of Dollars simply based on FDA recommendations, world follows and closely watch USFDA protocols. One SCAM company like SRPT cannot decide the fate of FDA.
What a tool! Man, you show up here and spew nothing but unintelligent garbage. Like you know jack. In fact you are a wuss. Putting down the boy's and their families makes you feel your da man? Your just one sick puppy.
That would be great.
Especially if he hasn't progressed to point where the drug is no longer effect.
How does Garabedian and board of directors sleep at night?
Their decision to go it alone to maximize profits has had a rather high cost to some.
How does Margaret Hamburg and the doctors in FDA's Neurological Products sleep at night? How do the folks at University of Leiden who out-licensed a patent that caused the development of a safe and effective drug for exon 51 sleep at night?
Pasteur and/or Ru I am not certain how you sleep at night.
And the effected boys probably don't sleep at night because of the steroids!
We have no way to predict what would have happened, if a partner had come forward or been engaged. GSK had some rights to the Wilton patents for years and apparently did nothing with them! It is not apparent whether the lack of reward in the Drisapersen trials is due to the drug or GSK screwing up the trial definition.
Partnerships usually don't leave the small guy in control. There are quite likely larger targets in Splice Switching than duchenne. Avi/Sarepta picked a target due to a dedicated Australian and its almost managable size for a small company.
Pasteur- lower extremity muscles may be too fibrotic by early teens for DMD boys like Austin to walk again, but upper thoracic (diaphragm, lungs, heart) might still have enough salvageable dystrophin fiber. Recall, SRPT data has shown favorable PFTs and that is why FDA wants an older & non-ambulatory group to be studied. Ultimately, the clinical benefits in the approved drug label may different for age & preexisting severity of the DMD patient.
Pasteur, you continue to harp on CG and the board about going I alone, and that they need to partner. What does a partner bring to the table to add value? Absolutely nothing. You have not come up with anything concrete to support your position.