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Re: Vango54 post# 105995

Tuesday, 05/23/2017 8:03:39 PM

Tuesday, May 23, 2017 8:03:39 PM

Post# of 458801
Rare diseases without treatments available can be approved early. The phase 2 trial for rett syndrome will be double blinded placebo controlled. Even if a phase 3 is required, a 3 month trial plus another 3 month trial is 6 months.

With Alzheimer's, a phase 2/3 will take more patients than the rett trial. The patients will start as enrolled. If it takes 6 months to fill a multi continent trial, it will take a year for the last patient to reach 6 months. There may be a line up of trial participants for AD due to the safe nature of this drug, but with the parameters being created...they will need to go through a lot of possible candidates who qualify. Then a lot more evaluation of data and more rigourous testing will be needed.

With rare diseases that have physically noticeable symptoms such as seizures, just a reduction in seizures would be an easy end point to quatify.
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