It was the day before a long weekend, and we were supposed to leave that afternoon for a family road trip we’d planned six months earlier. Six hours in Jude’s life in April 2026, clockwise from top left: Jude tries on glasses in the morning, is wheeled away in…
Oak Hill Bio has secured $32.5 million in financing to advance rugonersen, an experimental treatment for Angelman syndrome once in the pipeline at Roche, into late-stage clinical testing. The planned Phase 3 trial was dubbed pivotal by the developer in a company press release, which called rugonersen…
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…
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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