ASF and FAST Unite for Legislative Working Group
FAST, the Foundation for Angelman Syndrome Therapeutics, and ASF, the Angelman Syndrome Foundation, announce a collaboration for a legislative working group focused on furthering the awareness and treatment of Angelman syndrome through legislative advocacy. The legislative working group will continue to work with the Everylife Foundation and focus on three critical rare disease initiatives, including Speeding Therapy Access Today (STAT) Act, Newborn Screening Saves Lives Reauthorization Act and the Rare Disease Congressional Caucus.
- The Speeding Therapy Access Today (STAT) Act is a bipartisan bill established with the rare disease community’s input to improve the development of and access to therapies for the rare disease community. Most notably, the STAT Act would create an FDA Center of Excellence for Rare Diseases.
- The Newborn Screening Saves Lives Reauthorization Act will continue federal programs aimed to provide assistance to states. The Newborn Screening Saves Lives Reauthorization Act is designed to improve and expand newborn screening programs, support parent and provider education, and ensure laboratory quality and surveillance for newborn screening.
- Established in 2010, the Rare Disease Congressional Caucus is a bipartisan, bicameral platform for discussing critical policy issues. It provides rare disease patients with a voice on Capitol Hill through quarterly briefings that address issues impacting the Food and Drug Administration (FDA), the National Institutes of Health (NIH) and legislation affecting the rare disease community.
FAST and ASF will further champion the overall mission of advancing the treatment of Angelman syndrome through education, research and support for individuals with Angelman syndrome.
“FAST is pleased to partner with the Angelman Syndrome Foundation to further governmental outreach,” said Lauren Hoffer, director for the FAST Board of Directors. “Together, we will drive policies that fund Angelman syndrome medical treatments and research; meanwhile, improving the lives of our loved ones. FAST and ASF are stronger together.”
Amanda Moore, chief executive officer for ASF added, “We are thrilled to collaborate with FAST to improve policies and systems that will positively affect the Angelman community. By working together on these vital issues, it will amplify our voices and make a lasting impact for current and future generations.”
About Angelman Syndrome
Angelman syndrome is a rare, neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene. It is estimated to affect 1 in 12,000 to 1 in 20,000 people globally. Individuals with Angelman syndrome have developmental delays, balance issues, motor impairment, and debilitating seizures. Some are unable to walk and most do not speak. While individuals with Angelman syndrome have a normal lifespan, they require continuous care and are unable to live independently. There are currently no approved therapies for Angelman syndrome; however, several symptoms of this disorder can be reversed in adult animal models of Angelman syndrome, suggesting that improvement of symptoms can potentially be achieved at any age.