https://link.springer.com/content/pdf/10.1007/s12325-024-02782-4.pdf?
Not sure if this has been shared yet but the reality is that with a new drug comes challenges and that in time challenges can be overcome.
The main hurdle was FDA approval to have an actual drug on the market to help a condition like Rett that had no alternative. The remaining hurdles are those for the families to jump and find a way through. For some the hurdles are high, for others lower and for some no hurdles at all, but for some they couldn’t clear the hurdle.
The thing is this is real and it’s helping people. Hopefully those who don’t succeed will have 2591 in the future as an option
https://link.springer.com/content/pdf/10.1007/s12325-024-02782-4....
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