Newsroom Articles

Our new digital children’s book, Alexander’s Not-So-Secret Diary, brings this belief to life through the experiences of nine-year-old Alexander, who is living with Alexander disease (AxD), an ultra-rare, progressive and often fatal neurological disorder that affects approximately 1 in 1 to 3 million people worldwide.

For David and his mother, Melissa, moments of joy are an important part of life with Alexander disease (AxD), an ultra-rare, progressive and often fatal neurological disorder that affects approximately 1 in 1 to 3 million people worldwide.

Through their stories, families, advocates and experts paint a vivid picture of what life with Angelman syndrome looks like – the obstacles, the triumphs and the moments of strength that define their journeys. They share their experiences navigating the path to diagnosis, the hallmark signs and symptoms of AS and, most importantly, the incredible joy that their children with AS bring to their lives. 

Championing an investigational medicine to make a meaningful difference in the lives of those living with HAE