Rare Disease Week at Capitol Hill (February 27, 2024)
This week, I joined 800 people from all 50 states for Rare Disease Week at Capitol Hill ( https://everylifefoundation.org/rare.../rare-disease-week/ ) to meet with our representatives in Congress and advocate for legislation to improve access to treatments for rare diseases. Before today, I had never met with my Congressional representatives or their staff. Part of my motivation comes from what Scott and I went through when we realized how few effective treatments there were for pheochromocytoma/paraganglioma (pheo/para), his rare neuroendocrine cancer. Facing that reality, and seeing Scott realize it, is something that I can never forget. This is a common issue for people battling rare diseases. About 10% of the U.S. population has a rare disease, and less than 5% of rare diseases have an FDA-approved treatment. The only FDA-approved treatment for metastatic pheo/para, Azedra, was developed under the Orphan Drug designation but was recently discontinued by its manufacturer Lantheus du...