Hi Cure,

Check out the exciting research updates leading up to and at this year’s Global Science Summit!

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

Ultragenyx Pharmaceutical provided updates on its development pipeline, including investigational GTX-102 for Angelman syndrome at Analyst Day held, Monday, October 16, 2023.

We are pleased to have Ultragenyx present at our 16th Annual Global Science Summit where they will share their data and next steps for the investigational GTX-102 drug for Angelman syndrome to the community. 

Learn more about Ultragenyx’s updates:

Last week, Chief Operations Officer Ryan Fischer, Head of Regulatory and Policy Jennifer Panagoulias, and FAST Advisory Council (FAC) Member Karly Werner, attended the 2023 NORD Breakthrough Summit on behalf of FAST and Angelman syndrome. This event brought together over 900 rare disease advocates, experts, and stakeholders from around the world to tackle the most pressing issues facing the rare disease community. 

“The NORD meeting is a great chance to meet with other like-minded organizations working together to advance our causes - together, we are a powerful force for change in rare disease drug development and policy.” - Ryan Fischer, COO

NORD is dedicated to supporting education, elevating care, advancing research, and driving policy for rare diseases. 

Learn more about NORD:

The Global Angelman Syndrome Registry (GASR) team will be hosting a GASR Lounge at this year’s Global Science Summit in Miami, FL. 

A recently published case study showcases how the GASR used a novel approach to enable multiple language translations for its website, to build a robust database of caregiver insight on AS from around the world — thanks to members of our Angelman community for making these possible! The GASR is an invaluable resource to advance the search for therapeutics. 

Meet the GASR team at this year’s Global Science Summit! The deadline for in-person registration is this Friday, October 27, 2023.

Register now:

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