Can't rate something on a scale unless you define it. I think our very small, very preliminary PK phase analysis showed potential signal, and I suspect the larger full-dosing phase will show more. I'm looking forward to it, and the other two.
Our molecule is very likely acting on the human CNS, to some therapeutic benefit.
Let's face it. The Rett results could be a 10, where the girls assumed absolutely normal behaviors, with no more Rett symptoms. Were that to happen, the disbelief would be even greater. "Those results are highly questionable. No drug can restore normal CNS function. This needs to be tested more precisely, on a much larger population of Rett patients, overseen by an external monitoring agency."
Simply, there is great resistance to, and disbelief that there can possibly be a single, simple, orally-administered, safe drug that fixes CNS diseases. The experts, financial and medical, know.
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