Rett first has always been the plan to establish how the upstream S1R MOA operates using a genetic disease where the cause is known.
Back in 2015, when I established my first position (sub $1), I was deep into a dementia caretaker role for my dad (Lewy Body Dementia, alpha synuclein) and didn't understand the rational to focus on rare diseases. Flash forward to 2023 and we now understand the method to the madness in the high value of rare disease drug approvals.
Where things get weird for me is what happens to rare disease drug pricing when it's approved for a large indication like AD? I assume the price drops for those with the rare disease indications too.
Not suggesting that at all. That was a bit of a history to explain why pediatric Rett has not yet completed and to show that it was intended to be done by now.
That would have generated a revenue stream and validated the MOA.
The post was a response to the complaint about Missling lacking focus and not having anything approved yet.
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