Importance of the trial results & challenges, page-10

Agree Gas. I think a key step is approval from the FDA based on RBSQ as we know they can hit that in P3. Maybe this is where the market apprehension comes in. Previously agreed with the FDA that MBA was the measure, if we had have hit that then we would be expected to have no issues with approval for P3. Now the change in measure needs FDA agreement adding to uncertainty. I think the Rett community will lobby hard to get this through. FDA approval of P3 on RBSQ will then de-risk Trofinetide for Big Pharma as a likely path to approval.

This is the only issue I can see in not having a step change in SP on the current news. Maybe FDA P3 approval will generate some excitement.

Just picked up on page 7 - "In a pivotal trial, Neuren intends to use dosing that will aim to achieve similar exposure in subjects regardless of their weight", so they are all over my above comments regarding dosage.

I would love to see some raw data!