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Dan Gallo

Dan Gallo

Sep 3, 2026

LaurenHOST
1:28
So before we get into those nitty gritty details, give us kind of a brief overview of what the Developmental Milestone is.
1:37
Sure, happy to.
1:37
So our ultimate goal here with this study is to help accelerate drug development for individuals with PMS.
1:43
We want to be able to collect data that can help inform clinical trial outcomes that could ultimately support an approval while recognizing and helping to overcome some of the challenges that often in rare diseases when you're trying to develop a treatment you might face.
1:58
So in order to do this, we understand that the FDA has certain requirements for drug approval, and these can include that clinical trials be adequate, well-controlled studies that ultimately have to demonstrate what regulators call substantial evidence of drug effectiveness and safety.
2:16
In these types of studies, we have to show that the drug's benefit is clinically meaningful and that the changes seen are statistically significant.
2:23
And so designing these kinds of studies can be a challenge, especially in rare diseases, for a number of reasons.

16 MINS LATER

LaurenHOST
18:41
So what are you hearing from families about this part?

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