The CFF believes that other mutations "may" benefit from this drug....though it's mechanism works by increasing the number of d508 CFTR at the cell membrane as well as opening the channel (corrector and potentiator)...per what I've learned, they've studied hundreds of thousands, if not millions, of compounds to find VX-809. I'd like to know what the "compound" is, but we're not privy to that info. <img src="i/expressions/face-icon-small-sad.gif" border="0">
CFF scientists and doctors believe that an overall percentage of functioning CFTR could be as low at 15% for CF symptoms to subside significantly. Crazy!! Once a drug makes it to phase 2, it has a 50% chance of FDA approval....