I wasn't saying it would serve as particulaly regulator approvable standing on its own (though I personally believe regulators should become more flexible and speedy - 1 year waits for EMA to review data is crazy). However even in open labal access ad it would seem quite compelling if it was pre-specified that col24a1wt blarca patients would do better than non col24a1wt patients and indeed it then happens again one year on and with stat sig p value. Patients would not know their col24a1wt status and it could be easily arranged that the clinicians can not know either so double blind.
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