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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.

• May 5, 2025 Eric Crombez, M.D. Appointed to Massachusetts’ Rare Disease Advisory Council Dr. Crombez shares his perspective on what keeps Massachusetts on the leading edge of science and rare disease research Read more >
• March 10, 2025 Advocacy Guest Post: GSDIa Treatment Quest GSDIa advocates share about the historic support the community has provided for gene therapy research. Read more >
• February 26, 2025 Advocacy UltraAdvocates: Leading change for rare disease communities Uniting for healthcare improvements and reform. Read more >