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Hi ww,

completely agree with you on this one: in fact the trial will probably be adjudged a success even if there are "only" physiological benefits.

This is also true in the Fragile-X case: there are physiological aspects which have been successfully treated in the mouse models, so there should be clear indications of efficacy.

Perhaps as a results of learning from earlier trials, there are plenty of "objective" indicators in the trials too. Trial subjects spend a substantial time in hospital in outpatient services.

My understanding is that trial subjects are not required to sign any sort of non-disclosure agreement. I've been keeping an eye out in the blogosphere but can't see anything obvious out there. I've also been trying to "read the tea leaves" in the clinicaltrials updates, but there's nothing conclusive there yet either.

As a caveat, though, we should be careful about the power of wishful thinking. Until the results come out, there's a risk that anecdote overpowers science. Unless the results are spectacular and incontrovertible. And, while I try to avoid the trap of wishful thinking myself, that's my dream.