Ultragenyx Pharmaceuticals and Ionis Pharmaceuticals recently shared updates to the Angelman syndrome community on their plans for a Phase 3 trial or on their Phase 1/2 clinical trials for Angelman syndrome.

Read Ultragenyx’s update:

Read Ionis’ update:

For more information, we invite you to tune into the LIVESTREAM updates at the Angelman Syndrome Foundation (ASF) Family Conference on Wednesday, July 24th starting at 3:10pm ET. To access the link, click here.

We are excited to announce a new publication from Dr. John Marshall at Brown University made possible by FAST funding. The project started in late September 2022, aiming to modulate synaptic function with two novel compounds, Syn3 and D-Syn3.

This research represents a possible small-molecule approach (Pillar 3 – Downstream Targets) to potentially address the synaptic dysfunction observed in AS as a result of the loss of functional UBE3A in the brain.

Learn more:

Stephanie Ho and Fred Cheng of Hong Kong received the news last December that their son Asher lives with Angelman syndrome. They immediately began to find ways to help him – one of those ways was to host a charity concert, Angels Within Us, which took place on July 7th in Hong Kong.

This sold-out concert brought together more than 1,000 attendees! Thank you to everyone who worked tirelessly to make this special evening a reality in honor of Asher! 

Read more and check out photos:

Last Saturday, Payslie’s Pals of Fall River, Wisconsin, migrated to a local park for the 3rd annual cornhole tournament. This local fundraising event raised over $15,000 (their best year yet!) in support of FAST’s mission. Many thanks to Payslie’s parents Sean and Jena Berndt, along with all of their family and friends, who played for Payslie, and all who live with AS. Your continuous support for our community is so appreciated.

Read more and check out photos:

The first Latin American Angelman Syndrome Congress, organized by FAST LatAm, will be held at the Universidad Javeriana in Bogotá, Colombia on August 29-30, 2024. This event is a unique opportunity to connect and advance knowledge about AS in Latin America. We invite the global community to join the event in-person or virtually. The event will have simultaneous translations between English and Spanish. 

Learn more and register:

Thanks for reading, and please share this email with anyone in your network who would be interested in receiving our updates.