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Re: XenaLives post# 180836

Friday, 02/08/2019 9:32:48 AM

Friday, February 08, 2019 9:32:48 AM

Post# of 458250
Context, I think it was a question Missling wanted asked:



Jotin Marango

Congratulations on the enrollment progress. My question is to Christopher are about the Rett program. It's a very interesting new indication which is getting hotter out there. So is there one functional parameter or perhaps the constellation of parameters that are thought to be clinically meaningful in assaying improvement in the disease. Perhaps there are several are some of them more clinically meaningful to the patients. And then lastly, the important part of the question where do you think regulators are in understanding and then having consensus on what is important in evaluating a disease? Thank you.

Christopher Missling

Good questions, regarding the Rett indication. I will start with the second question, first. So there has been a response from sponsor with proposed Phase 3 design [technical difficultly] Kaufman which is now our Chief Medical Officer and the [indiscernible] consist of a group of behavioral and composite questionnaire of the symptoms of the Rett's pathology. We do know that the Rett pathologies many folds in its pattern. There is a not a unifying feature of these disease. I give you just a few examples, one of them is cognitive impairment. The other one is seizures, the third one is motor impairment and the fourth one is anxiety. So in all of these four features of phenotypes are adequately addressed in this RSBQ, Rett Syndrome behavioral questionnaire and so that now also answers your first questions I assume.








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