GTX-102, Ultragenyx’s investigational therapy for Angelman syndrome, improved cognition for participants in a a Phase 1/2 trial after up to three years of treatment. Most patients also saw clinically meaningful improvements in one or more key areas, including communication, motor function, sleep, and behavior. The data support starting…
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Rebecca Burdine, PhD, a professor at Princeton University, received two awards for her contributions to Angelman syndrome research. The Angelman Syndrome Foundation awarded Burdine the 2024 Harry and Audrey Angelman Award for Meritorious Service. The award, named for Harry Angelman, the doctor who first formally described Angelman syndrome,…
Prior to receiving our son Jude’s diagnosis of Angelman syndrome last April, when he was just 16 months old, we beamed with pride that he was a social butterfly who brought joy to everyone he met. Jude’s cheerful personality has always drawn attention. Passersby, checkout cashiers, and waitresses often…
Becoming educated and informed about Angelman syndrome is a good first step. Here you’ll find general information about the disease, including diagnosis, symptoms, and causes.
It helps to know others have been where you are now. Here’s a collection of our columnists’ words of wisdom to help you along on your Angelman syndrome journey.
There is no cure for Angelman syndrome yet, but there are treatments that can help manage the disease. Learn more about the approved and experimental therapies here.
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