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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 7, 2024 How you can inspire inclusion on International Women’s Day Read more >
• October 25, 2023 Advocacy A Rare Reunion 25 years after participating in a clinical trial, rare disease patients and families reunite with the researchers who developed a breakthrough treatment for a deadly childhood disease. Read more >
• September 21, 2023 Advocacy Achieving the Life-saving Potential of Newborn Screening After 60 Years of Newborn Screening, Political Leadership is Needed to Ensure Rare Disease Diagnosis Keeps Pace with Science Read more >