Angelman Syndrome (AS) research hub

An Open-Label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of RO7248824 In Participants with Angelman Syndrome

This study is looking at a new medicine called Rugonersen for people with Angelman Syndrome. It aims to see if the medicine is safe, how well the body handles it, and what effects it has. We are also checking for any side effects.