Long-term data from a clinical study suggest that the experimental treatment GTX-102 (apazunersen) is associated with continued improvements and developmental gains in children with Angelman syndrome, with results from a Phase 3 trial testing the therapy expected later this year, according to an update from developer Ultragenyx…
Discussion
These days, when friends and family ask about my 3-year-old son, Jude, who lives with Angelman syndrome, my answer comes easily. I tell them about his sweet, affectionate nature, like how he leans into us for cuddles and loves giving high-fives. I talk about his interests, like his newfound…
Surgery aimed at correcting strabismus — a condition in which the eyes are misaligned and do not point in the same direction, causing vision problems — is usually successful in children with Angelman syndrome, according to a new study led by scientists in China. The one-year success rate was…
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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