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
View all posts >-
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 >
-
February 14, 2025 Advocacy Raising a son with Angelman syndrome Insights on the family impact of Angelman syndrome from a caregiver conversation Read more >