The Next Frontier in FAST Funded Research

For 12 years FAST has been working diligently to fundraise, identify research opportunities, and wisely invest in science in order to develop real and effective treatments for our loved ones living with Angelman syndrome (AS). Thanks to your fundraising dollars, FAST has funded important pre-clinical work, developed much-needed outcome measures, and played an integral role in forming a pre-competitive space for pharmaceutical companies and scientists to share their findings to further advance AS research. We are proud that the Angelman community now has two gene altering treatments in clinical trials as well as one down-stream targeted treatment option.

FAST thanks you, our dedicated and determined community. Because of your tireless fundraising efforts, FAST has been able to commit $20 million to meaningful AS research grants that have accelerated the trajectory for a viable therapeutic in Angelman syndrome. You have brought us to where we are today. You are truly amazing!

However, we are not finished. We will not stop until every individual with Angelman syndrome has access to a meaningful therapeutic, regardless of genotype or age.

Yesterday’s fundraisers fund today’s science. Today’s fundraisers will fund tomorrow’s science. Tomorrow’s science will lead to the next groundbreaking approach in the treatment of Angelman syndrome. That is why fundraising for FAST is still imperative.

Here are the incredible research grants your fundraising dollars have developed in 2020:
Pilot feasibility of an enzyme replacement therapy for Angelman syndrome
Rodent Studies of Non-Snord 116 Paternal Ube3a activation
FAST Infrastructure grant to develop a core AS-specific facility as a resource for developing and evaluating various treatments for Angelman syndrome
– A human platform to efficiently study the most common genotype, and the various Class 1 deleted genes in Angelman syndrome
– The first Angelman syndrome specific communication measure developed by AS families and caregivers called the ORCA (Observer-Reported Communication Ability) outcome measure
– A Biorepository of Angelman syndrome IPSC lines and brain organoids
– The evaluation and utilization of the Actimyo device, a wearable to assess tolerability and characterization of Angelman syndrome specific gait patterns
Generation and characterization of a new AS mouse model that recapitulates the large deletion of human 15q11-q13 and a control line that carries a deletion of all genes in 15q11-q13 except UBE3A
– Characterization and assessment of the Angelman syndrome pig model to develop, test, and validate potential AS therapeutics
– Support various other countries in their work to identify and support individuals living with AS, like the Genetic Diagnosis Campaign in Argentina

Now what? FAST continues to invest in new key research opportunities. In order to invest, we need you to help us keep fundraising! Our community has two main opportunities to get involved. The first, the Cure Angelman Now (CAN) Community Fundraising Challenge has been an incredibly successful resource for our community to fundraise throughout the year. The 2021 CAN Community Fundraising Challenge will kick off early next year with our new platform, Just Giving.

The second opportunity for our community to support AS research is the virtual “paddle raise” during our Virtually Unstoppable mini-Gala on Saturday night, December 5th. In fact, you don’t need to wait until the mini-Gala to donate. You can donate now! Donations made between now and December 5th will be included in the scrolling sidebar throughout the mini-gala. You will be recognized for your donation in real-time during the fundraising event!

In the last three years the FAST community has raised $2.8 million through the CAN Community Fundraising Challenge and $9.9 million during the annual paddle raise at our yearly Summit & Gala. Again, you are truly amazing!

Thanks to your fundraising efforts, FAST will continue to fund groundbreaking AS science and further innovative discovery to work toward brining therapeutics to all individuals living with Angelman syndrome. Because today’s fundraisers will fund tomorrow’s science. Tomorrow’s science will lead to the next state-of-the-art approach in the treatment of Angelman syndrome.

Let’s #CureAngelmanNow

 

 

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