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Re: raja48185 post# 358901

Friday, 04/29/2022 11:10:14 PM

Friday, April 29, 2022 11:10:14 PM

Post# of 462338
They will file for adult rett approval. There is zero risk to the company with EXCELLENCE already in flight and reading out by year-end. The worst case, short-term scenario is a CRL indicating they need more data or population, which they have in flight and pre-negotiated with agency via EXCELLENCE and its now 85 patients, ages from 5-17 years old…this data set building on the prior US Rett and AVATAR studies. I fully expect they go for it since they lose almost nothing in trying.
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