Endpoints chosen in a trial are very important. Blood tests as...

Endpoints chosen in a trial are very important. Blood tests as hank alludes to are usually indirect or surrogate markers which have their limitations. You need to show that the blood test result clinically correlates to a benefit to the patient which can be difficult in neurological conditions. IMHO a patient or caregiver assessment directly relates to a perceived benefit by the patient or carer who are monitoring the patient 24/7. The placebo arm corrects for those replies hank indicates are just ticked off due to lack of interest. There was a difference which was stat sig so all good.
The endpoints chosen for Phase 3 IMHO are the best available for assessing efficacy of an intervention in Rett’s.