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Finding the best metrics for pediatric neurogenetic trials

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Science beakers on a lab table.

About this webinar

Neurogenetic disorders that impact how the brain functions affect up to three percent of children, but available evaluation tools aren’t sensitive enough to detect change in their developmental skills. This poses a challenge for clinical trial sponsors who are trying to assess potential treatments. In addition, detailed natural history studies haven’t done enough to characterize the effects of age and the stage of disease of neurogenetic disorders. 

Recent research has been calling for new and improved tools to test treatments of all pediatric neurogenetic diseases — something researchers and trial sponsors are eager to see. Join experts for a one-hour webinar that will help you better understand the challenges to treatment in these trial settings.  

You’ll learn:

  • Why available evaluation tools may not always be effective in clinical trials

  • The advantages of precise tools and metrics in developing clinical trial protocols

  • Why Growth Scale Values (GSVs) are a better metric for measuring developmental change during trials 

Have you seen our webinar?

Learn more about the importance of natural history studies in clinical trials, the challenges of testing children with genetic disease and much more!

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Speakers:

Elsa Shapiro

Elsa G. Shapiro, PhD, ABPP

Professor of Pediatrics and Neurology, Division of Pediatric Behavioral Neuroscience, University of Minnesota, Minneapolis, MN.

Julie Eisengart

Julie Eisengart, PhD, LP

Pediatric Neuropsychologist, University of Minnesota

Paul Harmatz

Paul Harmatz, MD

Pediatric Gastroenterologist, UCSF Benioff Children’s Hospitals

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