That's well stated. The question may be, will the company approach the regulator based on success in adults as well as other results suggesting benefits and hopefully gain a conditional approval, or will they wait for new trials that better demonstrate the benefits. The conditional approval would permit the company to market the drug, but require a confirmational trial. Of course if they don't submit, they must do further trials if they're going for approval in Retts. The drug could be approved in Alzheimer's, or other disease, and off label use in Retts would be possible.
I'm of the believe that they're going for AD first, once that's achieved they'll have the funds needed to either move forward in other indications, or run trials prior to approaching the regulators in other indications. I believe that there are many people with diseases like Retts, Parkinsons, etc who'll get the drugs off label once it's been approved for AD or any other indication based on the trial data that's available to date.
Gary
Bullish