Ultragenyx Pharmaceutical Inc. announced earlier this month that the European Medicines Agency (EMA) has granted Priority Medicine (PRIME) designation to GTX-102 for the treatment of Angelman syndrome (AS). PRIME designation is a program in Europe run by the EMA to help developers of promising medicines have more frequent interaction with the regulators in an effort to get medicines to patients faster.

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This year’s International Angelman Day was filled with heartfelt moments where communities around the world showcased their support for loved ones living with Angelman syndrome in their lives. We are so proud of this community for your efforts to bring awareness, educate, and raise funds for AS organizations around the world. 

Read the IAD round-up:

Currently, there are 4 active human clinical trials for investigational treatments for Angelman syndrome – two antisense oligonucleotides (ASOs) and two focused on downstream targets to treat symptoms. 

In addition, there are active observational studies where individuals living with Angelman syndrome can participate to help understand how the disorder looks over time, without a therapy, which helps us learn and better design clinical trials.

Learn more about trials and studies:

There is still time to register for the Inaugural Angelman Syndrome Congressional Advocacy Day in Washington, D.C. Please consider representing your loved one living with AS as their voice on the hill! 

Online booking for our discounted room block is closed. If you are still in need of a hotel room at the rate of $249/night, please contact events@cureangelman.org.

Join us in Washington, D.C. on March 6-7:

Rare Disease Week is quickly approaching, and we're thrilled to announce that both FAST and ASF will be joining numerous other rare disease groups on Capitol Hill in person. While we are in DC, we invite the Angelman syndrome community to register for informative sessions with the NIH, Everylife Foundation, and FDA and help spread awareness to the 7,000 rare diseases worldwide.

Learn more about Rare Disease Week: