Foundation for Angelman Syndrome Therapeutics

FAST is the leading patient advocacy organization working to cure Angelman syndrome. As the largest non-governmental funder of Angelman syndrome research in the world, our goal is to drive forward transformative research and development programs as quickly as possible for those living with Angelman syndrome — regardless of age or genotype.

What We Do
We set the agenda for the therapeutic landscape for AS and help to accelerate it, from funding promising research at the academic level all the way to starting companies; create the necessary infrastructure outside of drugs and their development, from projects like our global registry and newborn screening to preparing for regulatory approval processes and advocating for insurance coverage; and we activate and educate those in the worldwide AS community interested in and committed to clinical trials, and what the future of drug development will be for our loved ones.

At FAST, we believe we got lucky with our gene.
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.

Angelman syndrome (AS) is a rare neurogenetic disorder that affects about one in 15,000 people, or approximately 500,000 individuals worldwide. Some say it could be as frequent as one in 8,000 people, or over 1,000,000 individuals worldwide.
People living with AS have a genetic difference on chromosome 15. People with these chromosomal changes do not produce any, or remotely enough, of something called UBE3A—which is a protein involved in brain functioning that helps us walk, talk and perform many other everyday tasks.

As a result, children and adults with AS typically have balance issues, motor impairments and can have debilitating seizures. Disruptive sleep is often a serious challenge. Some never walk. Most do not speak. Individuals with AS have a normal life expectancy, but require continuous care and are unable to live independently.

This is life today for people living with Angelman syndrome.

But it won’t be for long.

Scientists believe that AS is now the neurogenetic disorder with the greatest potential to be cured. FAST is the reason why, and the force making it happen.

Grants
Unlike other organizations, we do not have a research deadline, but instead maintain a rolling application process—enabling us to scoop up promising ideas as soon as they surface, while being proactive and recruiting the best in the world with novel ideas to work on Angelman syndrome. Each proposal is sent to three members of our Scientific Advisory Board (SAB), made up of 25 individuals who are each subject matter experts in different areas. Currently, FAST has over 20 research grants that are active and ongoing in different research facilities around the world.

We invest in a myriad of projects related to supporting all of the work that has to happen around drug development, both before and after, to ensure that it moves safely and quickly. These efforts, which fall under our Pillar 4 work, includes: Creation of animal models and cell lines for each genotype; the Global Angelman Syndrome Registry and its related Search & Rescue operation; efforts at newborn Screening, which will give us the true incident rate of AS, which helps pharmaceutical companies estimate how many individuals could potentially benefit from treatments being developed; the Angelman Syndrome Biomarker and Outcome Measure Consortium (ABOM), which is devoted to achieving alignment on endpoints and biomarkers, which are required to accurately measure the effect of a drug has on patients in clinical trials.

And the jewel in our Pillar 4 crown: The Rush Pediatric Neuroscience F.A.S.T. Center for Translational Research: Global headquarter for training individuals in how to run neurogenetic clinical trials and deliver innovative interventional therapies.

What distinguishes FAST is our assertive approach to funding and dedication to nurturing collaboration among families, communities, scientists, clinicians, and industry stakeholders — all with the ultimate goal of bringing practical treatment into current medical practice as safely and quickly as possible.
Crucially, the engagement of pharmaceutical and industry partners plays a pivotal role in ensuring that potential therapies do not stall during the research phase, particularly in the challenging “valley of death” of drug development. FAST actively supports numerous clinical and nonclinical development initiatives undertaken by various pharmaceutical companies.

FAST is deeply invested in bringing promising interventions into current medical practice, as safely and quickly as possible—and we know scientists are not the only ones who will make this happen.
To this end, FAST is driving translational research and supporting the advancement of therapeutics for animal models to humans living with Angelman syndrome.

The goal is to advance as many promising therapeutic strategies as possible for Angelman syndrome, regardless of the obstacles, to find the best treatment options for our loved ones, for all ages and for all genotypes.

Supporting FAST’s mission can take many different forms.

* Donate Online & Other Ways to Give
* Family Fundraising
* Investing with FAST

Regardless of how you join this effort, you will help us continue our work to bring life-changing therapeutics to every individual living with Angelman syndrome. Our goal is to successfully complete this race—and no longer be necessary—by finding a cure.

CONTACT FAST:

PO Box 40307
Austin, TX 78704
Phone:
(630) 852-FAST

Fax:
(630) 852-3270

Toll Free:
(866) 783-0078
Email: info@cureangelman.org