I would think (desperately hope) that Rett is long approved before this "ultra-rare" disease trial is even started (recruited).
With second Rett trial to disclose data this year, I would hope FDA approval for adults is received by midsummer 2022 at the latest.
Ultra-rare wouldn't likely even be recruited at that point. That is, if it even gets started. The tease has been running for what, a year now?
Actually I don't see the point of ultra rare strategy. I would prefer we start a trial for epilepsy. Weren't Rett and Fragile x laying the groundwork for that?
And with all that data from Wayne state, whatever happened to MS?
Or better yet, a cardio trial?
The fishing for minnows strategy could use a little explaining in my eyes, when there are so many nice marlins swimming about to catch.