So I just checked Sarepta's website and confirmed what wam said: their phase II trial enrolled 12 patients.
In three separate strands.
Kind of makes 48-60 patients split between placebo and 2566 look overdone.
Oh and wam: Rett might be 1 in 10000 but fragile-x is 1 in 4000 if I recall correctly.
It's a long way to go and while I'm enthusiastic I'm probably in the middle of the TonyIndo - cortinaboy scale.
I'd guess Serapta is about a year on (if not more) from where the 2566 trial is now. It does raise the question of whether 2566 would require longer term efficacy results (the sarepta trial went as long as 74 weeks) to get approval.
rett vs dmd, page-2
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