As good as it gets with observation based scoring and never seen better in Rett.
I think with a rare orphaned disease this is good enough even without a recognised strongly correlated objective biomarker, cause no one knows of one.
It would have been even better with a recognised and strongly correlated objective biomarker. I think the market sort of automatically is looking for that, but I think we got this especially once EXCELLENCE readouts.
IMO we need to wait for EXCELLENCE before an approval process kicks in.
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