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Re: CkFacts post# 144907

Saturday, 03/17/2018 8:29:54 AM

Saturday, March 17, 2018 8:29:54 AM

Post# of 462572

Many Orphan Drugs have been registered in 6-12 months ( A 2-73 has OD Designation for Rett Syndrome ) , and any even "promising " results will be strongly pushed/ supported by Rett Foundation.



Agree w/CK observation. Also, our trial/context for data-environment is moving under our feet as we go. New trials will be based more on the science of the treatment than in the numbers as from trials in the past. Success based on % recovery is of course always going to be important but when even a small % recover where zero have EVER previously recovered or shown improvement then understanding why based on science will be the trails focus, not % of base. Even the smallest victory on this field will be massive and will present the learning opportunity. The whole rule set and response to trials will be rethought over night. Trials will no longer be simple pass/fail events. In fact RWE may rule the day. Consequently Biotec investment becomes an entirely different win/lose proposition.

Like many here I am confident that SOMETHING is about to happen w/AVXL and it cannot be soon enough for me.
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