Saturday, October 20, 2018 10:50:21 PM
Doc.....you have been intimately involved with previous trials so I trust your analysis of the timeline of the RS trial. However, as we discussed sometime back, I think your timeline is somewhat conservative. Given the close relationship of the patients' families with the RS Foundation, the data from the Natural History Study, the dedicated Rett Syndrome Centers of Excellence and their experienced staffs, the administrative aspects of the trial should move expeditiously. On the regulative side, if the FDA uses the modern precision medicine model of seamless and adaptive trials, imho your more conservative timeline could be considerably accelerated. With notable improvement in the quality of life of the RS girls along with a good safety record, there will be powerful advocacy from parents and worldwide RS organizations to get 2-73 to market. We must remember this a very debilitating disease which affects these girls and their families from birth to death which can be over four decades......much longer than the average Alzheimer's patient.
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