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…
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The beginning of a new year is a hopeful and optimistic time. It’s symbolic of a fresh start, providing an opportunity to reflect on the past and embrace the future. Hope can be challenging to let in when caring for a person with Angelman syndrome, a rare neurogenetic disorder…
GTX-102, Ultragenyx’s investigational gene therapy for Angelman syndrome, improved cognition for participants in 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…
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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