Hi Cure,

Check out our updates around research, pharma, awareness, the FAST community, and the Global 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.

Community engagement with the Food and Drug Administration (FDA) is critical as potential therapies move through the pipeline toward regulatory consideration.  

We are excited to share that FAST has been invited to speak at the FDA’s Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products (OTP) webinar on Thursday, October 5th!

The webinar, Warrior Families: Advancing Regenerative Medicine For Angelman Syndrome Through Science, will begin with a presentation by Dr. Allyson Berent, FAST’s chief science officer, and will be followed by a panel discussion with fellow rare disease advocates, sharing their experiences with advocacy and contributing to therapeutic product development for loved ones living with rare diseases.

Learn more about the webinar:

Ultragenyx Pharmaceuticals (sponsor of the GTX-102, an Antisense oligonucleotide for the potential treatment of Angelman syndrome) has issued a letter to both ASF and FAST providing an update on their program. While they did not provide any timelines, they acknowledged actively working toward evaluating the safety and efficacy of GTX-102 in patients younger than 4 years of age, as well as those with non-deletion-type Angelman syndrome (i.e. mutations, imprinting center defects, uniparental disomy, mosaicism.) They also commented on individuals previously treated with other ASOs, and that they are still gathering the information necessary to determine how and when they could be treated with another investigational product.

Click here to read the letter:

FAST is joining the global initiative to bring awareness to Augmentative and Alternative Communication (AAC) as October is International AAC awareness month! Many individuals in our community use some form of AAC to help give their loved one a voice. 

Check out the current AAC app discounts in October:

The Granville Angels of Ohio, the Renner and Rees family, are joining forces to support the Angelman syndrome community. On September 16th, they hosted their 1st Annual “Cornhole for a Cause” fundraising event. This inaugural event attracted 32 cornhole teams and around 200 people in total, raising over $17,500 for Angelman syndrome. In addition, it also inspired others to show up in the community — local police and the fire department, area high school cheerleaders, the local hospital, and more!

Thank you to these two amazing Ohio families and their community for your support! 

Read more about the event and check out photos:

Reminder: FAST’s hotel room block at The Diplomat Beach Resort in Florida will end on Oct 6th which means the $199/night rate will also end.

Register for the Summit & Gala and book your hotel if you haven’t already:

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