Foundation for Angelman Syndrome Therapeutics

12/22/2025

Ionis Pharmaceuticals has shared a letter to the Angelman syndrome community on the ION582 clinical development program. The letter covers progress on the Phase 1/2 HALOS and Phase 3 REVEAL trials, as well as the recently announced CHAMPION trial to explore ION582 in additional genotypes.

Read the letter: https://bit.ly/48WCeNK

12/21/2025

One winter night. Full courts, full hearts. More than 150 friends came together at Exmoor Country Club and raised over $72,000 to move Angelman syndrome research forward for Ryan and every person living with AS.

Learn more about what inspired this event: https://buff.ly/AZYuurP

12/19/2025

At Drexel University’s Angelman Syndrome Awareness Night, families, athletes, and fans came together to celebrate connection, community, and hope. Led by Gabe’s parents, Linda and Joe D’Orazio, the event raised awareness and support for FAST’s mission to move research forward.

Read more about this incredible partnership: https://buff.ly/wWYfUnx

12/18/2025

If you missed the 2025 FAST Global Science Summit last month, this is the catch-up you will actually want to read.

There were over 30 presentations discussing insights and updates across translational research, caregiver tools, industry updates, and more. We have summarized each presentation into bite-sized chunks to help understand what you need to know.

https://cureangelman.org/summit-2025-event-recap

12/17/2025

The Seagos proved that fundraising doesn’t have to be complicated—it just has to come from the heart. 💙 Every family can make a difference for Angelman research, one idea at a time.

Read their story: https://buff.ly/srlt3F4

12/16/2025

🧬Gene therapy is moving fast. Access is not.

A recent 60 Minutes segment on million-dollar gene therapies is a tough but important watch, because it highlights a reality rare disease families face all the time: a breakthrough can exist, and access can still be out of reach.

That gap is why advocacy is part of FAST’s work now, not later. We can help accelerate the science, but families only benefit when the systems around it are ready too, including how coverage and affordability decisions get made.

FAST is proud to partner with the Angelman Syndrome Foundation through Hope in Action to push for urgency at the FDA, clear expectations for evidence that matters to families, and policies that support real access when new therapies reach the finish line.

Want to help close the gap between breakthrough science and real-world access? Join us at angelmanadvocates.org to sign up for action alerts and learn how to join us for Advocacy Day in Washington, DC next March.

A new class of life-saving drugs is helping children who once had no hope. But some carry a price tag of millions for a single dose, and the American health ...

12/13/2025

💙 Thank you.

To everyone who has donated, started a fundraiser, shared a post, or simply told someone about Angelman syndrome: your support means more than we can say.

You are helping move science forward. You are helping researchers stay focused. You are helping families feel less alone and more hopeful about what is possible.

You are part of the work that is pushing Angelman syndrome research forward every day.

12/12/2025

Help us secure as many State Proclamations as possible before International Angelman Day on February 15, 2026.

This opportunity is a simple action you can take that can have a powerful result for the Angelman syndrome community!

🔎Find your state’s submission deadlines and request yours today: https://angelmanadvocates.org/state-proclamations

12/06/2025

November was a month of big-hearted momentum for FAST. 💙

🏌🏻The Heath's Heroes community hosted the annual Hole-Out Fore Heath golf tournament that turned a day on the course into a show of love and support for families living with Angelman syndrome.

🎫 The Seago family launched Trips for Tripp, a one-of-a-kind fundraiser that gave supporters the chance to win a guided trophy hunt in South Texas, turning every entry into fuel for FAST-funded research.

🎾 The 3rd Annual Paddle Party for a Purpose gathered more than 150 friends, neighbors, and supporters for a sold-out evening of platform tennis, fire pits, and fun to advance FAST’s mission.

We are deeply grateful to the families who hosted these incredible events and for the hundreds of supporters who showed up to support FAST and Angelman syndrome research! 💙

12/04/2025

Trying to make sense of gene therapy for Angelman syndrome?

The American Society of Gene & Cell Therapy (ASGCT) has created an easy-to-read Angelman syndrome overview that walks through the basics of AS, how loss of UBE3A affects the brain, and the main gene therapy approaches scientists are studying to address it. It also covers challenges, clinical trials, and key questions to consider as families think about emerging treatments.

Access the resource: https://buff.ly/2wCFOL3

12/02/2025

The future of medicine looks a lot like this: bold scientists, powerful tools like CRISPR, and a world that refuses to accept “maybe one day.”

On 60 Minutes this week, CBS followed a group of high school students in Georgia who used CRISPR gene editing to design a new way to detect and potentially treat Lyme disease, and took their work all the way to the iGEM “science Olympics” in Paris.

Lyme is not Angelman syndrome, but the story is a powerful signal of what is possible when determined scientists have access to cutting-edge tools.

At FAST, this is exactly the future we are working toward. Gene therapy, CRISPR, and other innovative approaches are already in the Angelman syndrome drug development pipeline. When we invest in promising research and approaches, we are also investing in the next generation of researchers who are ready to move this work forward now and in the critical years ahead.

💙 𝐓𝐡𝐢𝐬 𝐆𝐢𝐯𝐢𝐧𝐠 𝐓𝐮𝐞𝐬𝐝𝐚𝐲, 𝐲𝐨𝐮 𝐜𝐚𝐧 𝐡𝐞𝐥𝐩 𝐟𝐮𝐞𝐥 𝐭𝐡𝐚𝐭 𝐟𝐮𝐭𝐮𝐫𝐞.

Your support today helps FAST:
• Invest in research that targets the root cause of Angelman syndrome
• Give scientists and early-career researchers the resources and data they need to move from discovery into real clinical studies
• Create more possibilities over a lifetime for people living with Angelman syndrome

Right now, your impact goes even further.
Thanks to an anonymous donor, any gift you make between now and December 31, 2025 will be matched dollar for dollar, up to $50,000.

👉 Donate here: https://bit.ly/48dHpKh

To compete at iGEM, a sort of science Olympics, teens at a Georgia high school set their sights on finding a better way to detect and treat Lyme disease. The...

12/02/2025

💙 Three ways to give to FAST on Giving Tuesday:

1️⃣ Donate on our website
Visit https://bit.ly/48dHpKh and make a gift of any size. Every dollar helps move the science forward.

2️⃣ Start a Facebook fundraiser for FAST
Create your own fundraiser, share why FAST matters to you, and invite your friends and family to give. Search for "Fundraisers" in the Facebook search bar. More details below.

3️⃣ Share the FAST Facebook fundraiser
You can still make an impact by sharing FAST’s Giving Tuesday fundraiser: https://bit.ly/4iBMaRo Every share helps more people see it.

Thanks to an anonymous donor, any gift made between now and December 31, 2025, will be matched dollar-for-dollar, up to $50,000.

Every donation, every fundraiser, and every share helps fuel research and build a future with more possibilities for people with Angelman syndrome.

Thank you for being part of this. 💙