As a caregiver, I understand that raising awareness about Angelman syndrome requires us to proactively educate the public. This can be done via social media or by organizing and participating in advocacy events. No gesture is too small. We’re encouraging our community to show support on International Angelman…
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Supporters are gearing up for International Angelman Day (IAD), a community-driven event held every February that aims to raise awareness for Angelman syndrome and honor those living with or lost to the rare neurodevelopmental disorder. The celebration also aims to promote Angelman research and education and mobilize fundraising…
The first patient has been dosed in a Phase 3 trial evaluating the safety and efficacy of GTX-102, Ultragenyx Pharmaceutical’s experimental gene therapy for Angelman syndrome. The global Aspire study (NCT06617429) is expected to enroll about 120 children and adolescents, ages 4 to 17, with Angelman…
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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