Hey Cure,

This year is flying by — we have just a couple weeks left until our 2022 FAST Science Summit & Gala in Florida! 

We want to see our whole FAST community loud and proud in Miami, so make sure to get your Gala tickets before our final deadline this Friday, November 18th! Get your tickets now if you aren’t registered yet.

Unable to join us in Miami? Join us virtually! Sign up now for the live stream of the Science Summit & Gala.

If you have updates you would like to be included in next week’s newsletter, please email us at info@cureangelman.org.

FAST’s strategic roadmap is laid out in 4 pillars:

1. Replacing UBE3A, the missing or non-functional gene or protein, into neurons of individuals living with Angelman syndrome
2. Activating the silent paternal copy of the UBE3A gene in neurons
3. Improving function/coordination of neurons at the synapse (downstream targeting improving the communication between 2 neurons)
4. Accelerating and preparing for clinical trials and drug approvals to ensure all individuals around the world are able to benefit from potential future therapeutics for Angelman syndrome.

The Angelman Syndrome Biomarker and Outcome Measure Consortium (ABOM) focuses on the 4th pillar. FAST's Translational Research Symposium agenda includes the first 3 pillars. Bringing both aspects of our mission together, FAST is hosting our 4th Annual Translational Research Symposium and ABOM Scientific Meeting on Thursday December 1, the day before the 2022 Global Science Summit and Gala.

This meeting is a full day of brainstorming, collaboration, and data sharing between the incredible professionals working on Angelman syndrome drug development including over 300 scientists, industry partners, and medical professionals. This team will all come together to advance biomarkers, outcome measures, and clinical trial design. This group is working to leverage real-world data from individuals living with Angelman syndrome that have been captured through many of the ABOM initiatives. The second part of the day will focus on translational research programs that are being executed in independent laboratories around the world so that all stakeholders at the meeting can share experiences, expertise and ideas to move each program forward faster than if any of them were working alone. Together we can #DreamBig and #cureangelman.

Each year this meeting has grown in attendance. Starting in 2019, there were 109 attendees, in 2020 it grew to 195, 2021 there were 254, and in 2022 we are over capacity and have 319 confirmed attendees! These are all professionals working hard to help our community reach our mission: Transformative treatments for Angelman syndrome. What’s clear is that industry experts are excited about Angelman syndrome and the possibilities. This effort is creating a platform that all academics, clinicians, and industry partners can leverage, and many other rare disease organizations are now modeling.

Click here to learn more about the importance of this work:

If you are an Angelman syndrome caregiver and a US resident, we need you to share your experience on the Caregiver Impact Survey – launching soon! 

The information collected will demonstrate the impact of AS caregiving in a way that previous research hasn’t captured. This is critical information that insurance companies and governments will need to cover the cost of a therapy. This study will also help communicate the benefits of AS therapies to policymakers, government agencies, and others who provide financial assistance to AS families. 

This survey will be housed on the Global Angelman Syndrome Registry (GASR) website. 

As we wait for the survey to launch, we ask everyone to ensure they have an account on the GASR website. 

Click below to access the registry so when the survey is active, you can start right away:

FAST France was formed in 2019 and is led by chairperson Anthony Moisan and Charlotte Préstat. This FAST Global organization is focused on 3 main objectives:

1. Translating Angelman syndrome information into French;
2. Bringing together key professionals to get properly set up for clinical trials in France;
3. Mobilizing private and public funding.

Click here to read more about FAST France:

Back in August of this year, we announced 5 new members of the FAST Advisory Council (FAC). Today, we are thrilled to introduce three more members who will help expand the FAC beyond science and research and focus on new areas to support FAST's development and organizational needs.

• Sue Edberg — former executive leader in the corporate and non-profit sectors
• Johnna Webb — former Controller of a local hospital and currently working in the hospital financial setting
• Karly Werner — attorney, Sr. Director, Government and Regulatory Affairs for Comcast

Since August, the FAC has been working on a robust policy strategy for advocacy and lobbying for the needs of the Angelman syndrome community. They are meeting with state officials and accelerating fundraising and awareness efforts within the community in the future.

Learn more about these new members:

October 29th marked the first annual Grayson’s Ghosts and Ghouls Costumes for a Cure, hosted by the Jacob family. Two weeks ago, the Austin community came together and raised over $420,000 to find a cure for Angelman syndrome!!!

“To say we are blown away would be an understatement”, say Ryan and Annie Jacob. “To see how many lives our kids touch and the incredible generosity of others is indescribable. Thank you to everyone for coming, donating, and supporting Grayson and others with Angelman syndrome. We are beyond thankful and appreciative of each of you, and can’t wait for the day G can tell you all himself!”

Click here to check out photos from the event:

Don’t miss your last chance to join us in Florida – get your tickets now!



As always, please share this link with anyone who would be interested in receiving our updates.