Ultragenyx announced the completion of enrollment in the Global Phase 1/2 Trial of GTX-102 in Pediatric Patients with Angelman syndrome. GTX-102 is an investigational antisense oligonucleotide delivered via intrathecal administration and is designed to target and inhibit expression of UBE3A antisense transcript (UBE3A-AS).

Their most recent press release shared that data from at least 20 patients enrolled is anticipated to be released in the first half of 2024.

Read the press release:

Join us next month for IAD! Let’s see how many buildings we can light up in blue, how much money we can raise, and how far and wide we can spread awareness to Angelman syndrome. 

Check out all the ways to participate:

Why research matters: Angelman syndrome needs our attention. Clinical research is the key to unlocking treatments and enhancing the quality of life for those impacted.

Spread knowledge: Share informative resources to educate and empower. Every share contributes to building a more informed and supportive community.

Support the cause: Consider donating to organizations helping to drive Angelman syndrome research. Your contribution can help fund critical studies, bringing us closer to breakthroughs.

Wear Blue, Glow, and Share Hope: On February 15th, wear blue and share your photos using #IADFeb15 #cureangelman #angelmansyndrome. Let’s create a wave of blue on social media, asking your local iconic buildings to be lit up in blue, symbolizing our commitment to awareness and change. 

Together, let’s turn International Angelman Day into a beacon of hope, advocacy, and progress! #DreamBig #CureAngelman

FAST and ASF are pleased to announce that we will be co-hosting the Inaugural Angelman Syndrome Congressional Advocacy Day March 6-7, 2024 in Washington, D.C.!

This advocacy opportunity is open to parents, caregivers, grandparents, researchers, clinicians, and friends of individuals living with Angelman syndrome who are willing to educate Congressional leaders and staffers to ensure that Angelman syndrome, while a rare disease, is forefront in their minds.

Learn more and register:

Save the date: November 8-9, 2024 for FAST’s 17th Global Science Summit & Gala in Orlando, FL. This is a robust weekend filled with knowledge, community, and celebration! You won’t want to miss it! 

Learn more about this year’s Global Science Summit and Gala:

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Email info@cureangelman.org with any changes to your contact information!

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