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.
Latest posts
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May 15, 2023 Advocacy Living with MPS VII Perspectives from rare disease caregivers and advocates Read more >
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April 10, 2023 Research The secret behind a rare disease blockbuster with global impact Partnering with patients to drive drug development Read more >
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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 >