If this is still relevant (http://www.graceforrett.com/rett-syndrome/r168x/nnz-2566-in-plain-english/) then it seems testing nnz-2566 on adults is a precursor to then testing it in the pediatric population. The article was written by Steve Kaminsky and Nancy Jones.
That would suggest that a successful result here sets up a similar trial for younger patients, and perhaps a wider Phase 3 trial focussed on efficacy? But I'm really shooting in the dark with this.
In terms of applications for Orphan and Breakthrough status for Rett, that will inevitably happen for 2566 if efficacy comes through.
If this is still relevant...
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