Re: Good news keeps flowing, along with some conflicting emotions - see Charley's Fund blog
Sad that all the exons need slightly different treatments. We are in a time which, a few years henceforth, will be looked back upon as one where some were lucky enough to be 'just in time' and others, tragically, will come 'just too late.' As you well know, I don't have to add. Hopefully the FDA is on board with proof-of-concept (is that the wrong term?) thinking: so that not ALL exon treatments will have to run thru a regulatory gauntlet as rigorous as Sarepta's first one was forced through. That will speed up conditional approval for the crucial other exon treatments to follow.