Roadmap to a Cure

For starters, Angelman syndrome is monogenic, which means we can focus our efforts on one place, and it’s also marked by imprinting, which offers us more strategies for fixing the mistake. But there’s a third feature of AS that makes it easier to work for treatments, and eventually a cure: it’s not a degenerative disorder. Various approaches, which have already been tested in animal models, are believed to have the potential to reverse the effects of the disorder in children, teens and adults.

The task before us at FAST is a daunting one: Can we change biological fate? How do you balance pushing hard at scientific and technological advancement while also keeping your loved ones safe? What will it look like to fix a genetic mistake in a person who’s already lived and developed for years with it?

We don’t know the answers to any of these questions, but we’re committed to finding out.

The only way to do something new and a little radical, though, is to do it responsibly. From here, you can learn about the broader landscape of drug development and how we are operating in it, as well as what the current field of AS drug development looks like:

• Drug Development 101

• The Current AS Pipeline