Take this FAST ACTION today: Parents, caregivers, and family members — Watch the EL-PFDD meeting and submit a comment.

This is your chance to ensure the lived experience is understood by FDA, companies, and by researchers in the AS space. 

Last November, Ionis Pharmaceuticals announced their phase 3 trial for ION582, called REVEAL, an investigational ASO for the potential treatment of Angelman syndrome. 

REVEAL is now listed on ClinicalTrials.gov. To learn more, join us at the community webinar hosted by ASF and FAST along with the Ionis team on Wednesday, April 23, 2025 at 1:00pm EST.  

Submit your questions and register below:

Oak Hill Bio enters into exclusive license agreement with Roche to obtain global rights for a Phase 3-Ready, Potential Best-in-Class Treatment for Individuals with Angelman Syndrome. 

Oak Hill Bio Ltd is a clinical-stage biotechnology company focused on acquiring and developing life-changing rare disease therapeutics. They issued a press release today along with a community letter that includes FAQs. 

Read the community letter and FAQs:

UBE3A protein plays a critical role in brain development and neuron maturation. Loss of function of UBE3A protein leads to Angelman syndrome (AS) while gain of function of UBE3A protein has been linked to an increased risk of autism spectrum disorders.  

This newly published review article authored by FAST-funded researcher Yu-Wen Alvin Huang (Brown University) examines the expression of UBE3A protein in neurons as well as other brain cells and highlights the involvement of UBE3A protein in different neurodegenerative diseases, including Huntington’s Disease (HD), Alzheimer’s Disease (AD), and Parkinson’s Disease (PD).   

A more comprehensive understanding of the influence of UBE3A protein on brain function and the development of neurological disorders outside of Angelman syndrome and autism spectrum disorders may provide new targets for therapies to treat Angelman syndrome and other diseases.   

Read more about this new article:

At a moment of unprecedented momentum in Angelman syndrome research and therapy development, we must not let up on either government or private investment.

FAST and ASF stand united in our commitment to drive research forward, accelerate therapeutic development, and ensure families affected by Angelman syndrome have access to the care they deserve.

Read the full response:

Bike 4 Briar is a CAN fundraising event pushing the limits on physical and mental endurance like never before. Riding in memory of John Nuck, grandfather to Briar (5) – who lives with AS, Todd Downey and Charlie Nuck are taking on a challenge of a lifetime to raise awareness of Angelman syndrome and funds for FAST along a 4,300 mile cross-country bike ride.

Their 56-day journey begins in Washington and ends in Maine, and we are asking the AS community to rally around these ambitious riders in a few different ways!  

Learn more and see how you can support this journey:

FAST is excited to announce three new team members to help us advance our mission. Please join us in welcoming Kelly, Sarah, and Amy to the team! 

🔷 Kelly Knee, PhD – Senior Director of Translational Research 

🔷 Sarah Traisman – VP, Head of Philanthropy Operations 

🔷 Amy Schamp – Senior Director, Strategic Communications  

Learn more about Kelly, Sarah, and Amy:

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