Spring Advocacy Update: Educating, Advocating, and Building Momentum
A productive spring for Hope in Action: all Angelman syndrome priorities included in House FY27 appropriations reports, plus a new DOD grant opportunity now open thanks to your advocacy.
FAST Brings Angelman Syndrome Community Perspective to FDA Rare Disease Roundtable
FAST joined 15 advocacy groups at the FDA Rare Disease Roundtable this week, representing the Angelman syndrome community as therapies advance from research toward real-world development.
Hope In Action: Angelman Community Delivers Big Wins in FY26 Fund Bill
Congress passed FY26 appropriations with all three Angelman syndrome priorities intact, unlocking $370 million in DOD research funding and advancing key FDA and NIH commitments.
Voice of the Patient Report Submitted: A Powerful Milestone for the Angelman Syndrome Community
The Voice of the Patient Report from the Angelman syndrome EL-PFDD Meeting has been submitted to the FDA, capturing families’ lived experiences to help drive meaningful treatments forward.”
MavriX Bio MVX-220 Update
MavriX Bio’s gene therapy MVX-220 receives FDA Fast Track designation, with its Phase 1/2 ASCEND-AS trial now listed and a community webinar set for October 9.
Ionis Receives Breakthrough Therapy Designation for ION582 in Angelman Syndrome
Ionis Pharmaceuticals’ ION582 has received FDA Breakthrough Therapy Designation for Angelman syndrome, a status reserved for drugs showing substantial improvement over existing treatments.
MavriX Bio Announces FDA Clearance of IND Application to Initiate First-in-Human Study of Gene Therapy for Angelman Syndrome
The FDA has cleared MavriX Bio’s IND application for MVX-220, an investigational AAV gene therapy for Angelman syndrome.
Progress for Angelman Syndrome Relies on a Strong NIH and FDA
Angelman syndrome research is at a turning point, and federal cuts could put that progress at risk. FAST and ASF are urging congressional leaders to protect the NIH and FDA expertise that helps move treatments safely to families.
RSVP for the January 2025 EL-PFDD Meeting
FAST and ASF will co-host a virtual Externally Led-PFDD Meeting on January 29, 2025, giving the Angelman syndrome community a direct voice with the FDA on therapy development.
The PFDD Survey is now open
The EL-PFDD Survey is now open, inviting parents and caregivers to share their experiences with Angelman syndrome to help shape the January 2025 FDA discussion.