
Dan Gallo
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Sep 3, 2026
Diving into the Developmental Milestone Survey with Dan Gallo and Tessa Clarkson from Jaguar Gene Therapy
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1:28LaurenHOST
So before we get into those nitty gritty details, give us kind of a brief overview of what the Developmental Milestone is.

Dan GalloGUEST
So our ultimate goal here with this study is to help accelerate drug development for individuals with PMS.

Dan GalloGUEST
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.

Dan GalloGUEST
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.

Dan GalloGUEST
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.

Dan GalloGUEST
And so designing these kinds of studies can be a challenge, especially in rare diseases, for a number of reasons.
16 MINS LATER
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18:41LaurenHOST
So what are you hearing from families about this part?