I am sure there have to be satellite CRO sites to support the parents and patients, collect data
Guessing the robust RWE will be a factor in market/patient acceptance. Trial news can cut both ways of course (positive/not positive) my guess is cautionary agreements will need to be established w/verification techniques.
I have been hoping that RWE allows Dr.M.. patients and the AVXL team to take the whole sp up/down control thing out of the hands of BP/WS.
What a fantasy, not only are pts getting effective treatment but the manipulators cannot do a thing about sp.