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FAST-Funded Communication Endpoint Leveraged for Prestigious FDA Grant

FAST, the Foundation for Angelman Syndrome Therapeutics, is pleased to unveil an FDA Patient-Focused Drug Development Grant that will evaluate the expansion of the existing Observer-Reported Communication Ability (ORCA) measure for use with other rare neurodevelopmental disorders.

The FAST-funded research team at Duke University was recently awarded the prestigious FDA Patient-Focused Drug Development Grant, designed to increase the ORCA measure originally developed to assess individuals living with Angelman syndrome. This team, led by Drs. Bryce Reeves and Christina Zigler will extend the existing ORCA measure for use with other rare neurodevelopmental disorders. The ORCA, funded by FAST in 2018, is currently being used in clinical trials and the natural history study to assess caregiver observations in receptive, expressive, and pragmatic communication abilities for individuals living with Angelman syndrome. 

"We are extremely grateful to FAST and to all the AS families who generously shared their time and participated in the studies to develop the ORCA measure. As in Angelman syndrome, families caring for children with other neurodevelopmental disorders have identified communication ability as a high-priority treatment outcome. By building from the foundational studies supported by FAST, our work to further expand the ORCA measure will help facilitate patient-centered clinical trials for individuals with a range of neurodevelopmental disorders, most of which are rare. By using a common metric to estimate communication ability, we will also be able to compare results for this important domain of functioning across clinical trials and patient populations," said Dr. Zigler.

"This award is a testament to the hard work and forward-thinking approach that FAST and the Duke team have taken to understand the unmet clinical need for those living with AS, as well as understanding the most important aspects of improvement in abilities that families and caretakers dream about for their loved ones. Families universally feel that improvement in communication abilities would be significant after a successful therapeutic intervention and this tool was designed, with FDA guidance, to accurately assess that in the Angelman population. We are so proud of this team," said Dr. Allyson Berent, chief science officer for FAST.

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