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
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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
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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
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