Research
Angelman syndrome is believed to have a high chance for a cure as scientists and researchers know which gene causes Angelman Syndrome. They have been able to reverse it in mouse models.
This is a picture of the Angelman Research Pipeline. There are multiple approaches that Scientists are focusing on that will benefit those living with AS. Currently, there are clinical trials being held throughout the US. Meaningful clinical impact has been seen.
Currently, the clinical trails that are happening use ASOs. Both companies are heading into phase 3. Watch this video to learn more about this approach!