Hi Cure,

For those in the United States, we hope you had a restful and meaningful Memorial Day weekend.

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

Some unfortunate news: PTC Therapeutics has decided to discontinue the development of this program. According to representatives of the company, who spoke with FAST and ASF last week, the decision was not related to the investigational therapy but based on their company's strategic prioritization of other programs. Both they and we are committed to finding an outside partner to move this asset forward.

We know this news is disappointing. As Dr. Allyson Berent, FAST’s chief science officer, always says, drug development can feel like a series of chutes and ladders, and it would be understandable to see this move — coming after our community watched this program progress in the Angelman pipeline for over 8 years — as a slide down. But that’s why we do what we do at FAST, and in moments like these we don’t rest — we double down on our energy. We are going to advance as many promising therapeutic strategies as possible for Angelman syndrome, regardless of the obstacles, to find the BEST treatment options for our loved ones, for all ages and for all genotypes. 

You can read the full press release from PTC Therapeutics here:

This week we are highlighting two newly published, FAST-funded publications on the Observer-Reported Communication Ability (ORCA) measure for individuals living with Angelman syndrome. Communication is essential to daily functioning and has been identified by caregivers as a top priority to focus on for treatment efficacy in clinical trials. 

There were some exciting findings! Read more to see how this tool has evolved, is now used in all of the active clinical trials for AS and how the FDA validated the work of the Duke team.

Read more:

Our next fireside chat is coming up, titled “UPD - ICD - Mosaic: Everything We Know (and Don't) About Minority Genotypes!”

Join us this Sunday, 6/4 at 12pm ET (New York), as Alana co-hosts this chat with Vlad Korotov and other UPD/ICD/Mosaic parents. They will sit down with Dr. Allyson Berent to have a conversation about AS minority genotypes.



Register now:

Did you know we have a FAST Shop? Head to our website to browse through the variety of apparel, drinkware, and accessories — for all ages. International shipping is available!

Save 20% on purchases June 1-5th!

Shop now:

Thank you to all of the families who entered to win the 2023 FAST Puppy Giveaway. The breeder reviewed the applications and selected Caleb and the Kovacs family. The Kovacs named the puppy Sunny and welcomed him with love this weekend. 

This breeder has continually supported the FAST community with her generous donations of a puppy to a welcoming Angelman syndrome family home. We appreciate her generosity! 

Stay connected for more opportunities in the future for your chance to win!

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