CAN Spotlight: Raye’s Birthday Fundraiser
Three-year-old Raye celebrated her birthday and Early Intervention graduation with a party that raised over $1,000 for FAST’s mission to find a cure for Angelman syndrome.
February 2025: Advocacy updates and actions to take
Despite the postponement of the EL-PFDD meeting, FAST and ASF continue joint advocacy work, from Congressional Advocacy Day to new toolkits and a rare disease policy letter to the administration.
FAST-Affiliated Research Program at UCLA is awarded $5.8M to advance gene therapy for Angelman syndrome.
UCLA’s Dr. Roger Hollis received a $5.8 million CIRM award to advance a stem cell gene therapy for Angelman syndrome, moving the FAST-backed program closer to a Phase 1 trial.
CAN Community Spotlight: Joelene
Joelene Wand, mother of Jude, shares how attending the FAST Global Science Summit renewed her hope, deepening her advocacy for the Angelman syndrome community in Australia.
Introducing the Innovation Seed Grant: Supporting Early-Stage Angelman Syndrome Research
FAST launches the Innovation Seed Grant, offering $10,000–$50,000 to help researchers gather early-stage data on bold, high-risk approaches to Angelman syndrome treatment.
FAST Germany’s Wachgeküsst campaign
FAST Germany relaunches ‘Wachgeküsst,’ aiming to raise €250,000 for Angelman syndrome research, building on a 2020 campaign that funded Munich’s first Angelman syndrome clinic.
Update on Advocacy Efforts and Ways to Get Involved in 2025
FAST and ASF kick off 2025 with advocacy updates, outlining ongoing FY25 congressional requests, key healthcare legislation, and ways for families to get involved this year.”
Ultragenyx Announces First Patient(s) Dosed in Pivotal Phase 3 Aspire Study Evaluating GTX-102 in Angelman Syndrome
Ultragenyx announces the first patients dosed in its pivotal Phase 3 ASPIRE study evaluating GTX-102 for Angelman syndrome, a major step toward potential treatment.
FAST Spain Awards First Research Grant
FAST Spain awards its first research grant to study two novel biomarkers—the endocannabinoid system and transcriptomic profile—to better track Angelman syndrome progression and treatment.
FAST and ASF Advocate for Crucial Legislation
ASF and FAST sign a joint letter supporting key legislation—the PRV Program, MVP Act, and Accelerating Kids Access to Care Act—to speed treatment access for the Angelman community.