>> How much have they really learned in the past 2 yrs in regards to inclusion criteria?
If you really think not much has learned in the past two years about trial designs from various Ataluren, Drisapersen, eteplirsen trials, well, I don't know what else to say except sorry I waited time. SRPT didn't have mid-scale manufacture ready until end of last year to earlier this year. Drisapersen ph3 failure changed many things - not interested in rehashing the whole thing on this board. I would guess PTCT would want to change certain things in their ph3 if they could.
I had worked on both "big" and "small" before, a lot easier in "big" disease unless in areas virtually no success in the past. Otherwise, you can come up with 10 designs by copying from someone else with minor changes here and there. Only those who never worked on these "small" diseases think they are easy.
