I know this has probably been discussed before but I find it absolutely incredible to consider the 2591 results in mouse models, especially in relation to Cognition/Learning & Memory and what it possibly means for neuro-degeneration in general, not just the specific rare diseases we are trialling.
Not only does it seemingly normalise the mice with the defective gene, if you look at the charts, it seems to improve cognition in the normal mice that are taking 2591, exceeding cognition of normal mice that are not taking 2591.
If that can be replicated in humans, the possibilities and ramifications are incredible to consider. I know JP has mentioned that they are not currently looking at "Autism" given the challenge in trials and pricing orphan and non-orphan drugs (amongst other considerations), but I am not sure that logic will hold if it proves to effectively treat neuro-degenerative conditions and even other neurological issues like concussion/TBI etc.
I have said it before but Ivermectin keeps coming to mind - it strikes me as similar in that the uses for it expanded beyond initial trials and it was found efficacious in a range of conditions, kind of like a pipeline in a drug.
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