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Rare perspective blog

Insights on our approach to collaborating with the rare disease community, supporting our teammates at Ultragenyx, and developing new therapies for rare diseases.

• March 22, 2023 Research Publication of the innovative research behind the discovery of GTX-102 for Angelman syndrome The decades-long work conducted at Texas A&M was featured in the March 2023 issue of Science Translational Research Read more >
• March 14, 2023 Advocacy VIDEO: This ‘Bootcamp’ Helps Parents Advance Rare Disease Research to Save their Children More than 100 nonprofit patient organizations have participated in Ultragenyx-led Rare Bootcamp events since 2017 Read more >
• March 9, 2023 Advocacy N-of-1 therapies: How clinical trials for a single patient can change the future Q&A with advocate Julia Vitarello on an emerging pathway for developing therapies for ultrarare diseases Read more >