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Re: falconer66a post# 132535

Friday, 12/01/2017 1:43:20 PM

Friday, December 01, 2017 1:43:20 PM

Post# of 518966


"Very similar with Rett syndrome patients. First, these are neurologically compromised little girls. Their ability, at least in some cases, to understand that pill they will be taking should suppress their epileptic fits and motor neuron hyperactivities and poor muscle control is minimal, at best. Hard to conceive how they could formulate the expectations of improved health required for the placebo effect to actually happen."

Pill? Falconer how about they arent even told they are taking a medication that is going to make them better......it is just administered to them by their parent.

See question #4 below at http://rettsyndrometrial.com/

4 ) Is your child with Rett syndrome able to take a small amount of liquid formulation (tablespoon) of investigational medicine, which can be mixed into juice or applesauce or other light food, once daily?
Yes
No
Don't know


Now if they are under a some sort of observation period documenting their daily disease symptoms and then a 4 year old is given a dose in a drink without knowing and improvement is observed (maybe drastic improvement in some cases), what is the parent/caregiver going to ask the FDA, Anavex, CRO to do at the end or during an unmasking of the trial?

I agree with Falconer that a placebo would not be applicable to a toddler, maybe candy but not a placebo.

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