On March 7, 2024, Angelman syndrome advocates from around the United States met in Washington, D.C. for the Inaugural Angelman Syndrome Congressional Advocacy Day. In preparation for the event, ASF, FAST and our partners, Faegre Drinker and Soapbox, led a […]
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FAST and ASF will be joining numerous other rare disease groups on Capitol Hill in person. While we are in DC, we invite the Angelman syndrome community to register for informative sessions with the NIH, Everylife Foundation and FDA and […]
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ASF and FAST are proud to be among over 160 patient advocacy organizations urging Congress to consider two technical corrections to address the unintended consequences of the orphan drug exemption of the Inflation Reduction Act that will help preserve hope and […]
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The past month has been a real rollercoaster for our family with many sleepless nights and a mix of emotions from uncertainty to anxiety due to Non-Convulsive Status Epilepticus (NCSE). I want to share our recent experience in hopes that […]
Read moreIn this exclusive Q&A session, we heard from a remarkable individual who wears multiple hats— a mother, a pediatric gastroenterologist, and a physician-scientist. She is on a mission to improve the lives of those dealing with neurodevelopmental differences. Kara Margolis, […]
Read moreNovember is Caregiver Awareness Month. To honor the loving, hard working, remarkable caregivers of the Angelman community, we’ve compiled a list of resources and services geared toward YOU, the caregiver. Even though caring for an individual with AS can be […]
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Ultragenyx Pharmaceutical provided updates on its development pipeline, including investigational GTX-102 for Angelman syndrome (AS) at Analyst Day held Monday, October 16, 2023. For GTX-102 the antisense oligonucleotide for Angelman syndrome, UGX reported data from the extension cohorts in the […]
Read moreRecruitment is open for the Aldebaran study, sponsored by Roche, to assess an investigational drug (called Alogabat) to potentially treat Angelman syndrome. While it does not involve replacing or unsilencing the UBE3A gene in individuals living with AS, it’s a […]
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