"By sharing my story, they not only learned about amyloidosis; they also heard about our community's desperate need for approved treatments and pledged their support"
I attended Rare Disease Week on Capitol Hill (February 29th– March 4th) as a Rare Disease Legislative Advocate for Amyloidosis and found this experience to be exhilarating and innovating! I was team lead for a small group from New York that included others with rare diseases. Our mission was simple and very effective: To educate Congress (Representatives and Senators) for the fight for life from the rare disease community. Of the 7000 rare diseases in the U.S. there are only 400 approved treatments and that has taken 30 years. Doing the math, a radically different approach to treatments for rare diseases needs to be implemented and become the new standard.
Our first day was the special day of February 29th, a rare day on the calendar, and was held at the National Institutes of Health (NIH). We heard from top-level scientists and doctors that shared some out-of-the-box thinking regarding rare diseases. They assured us that they are committed to implementing new research and policies to address the requirements of the rare disease community.
By Wednesday March 2nd we hit Capitol Hill with our teams to advocate for the rare disease community and what an exceptional successful experience our team had with the New York Congressmen. We met with NY Representative Tom Reed and Senator Charles Schumer. We also had meetings in 4 other congressional offices and in all of our meetings, they were very attentive to our stories and interested in our cause to increase legislation to accelerate research and enable quicker solutions for the rare disease community. This was such a positive day and I felt we made great strides in opening new channels for researching the much-needed solutions for rare diseases. By sharing my story, they not only learned about amyloidosis; they also heard about our community's desperate need for approved treatments and pledged their support.
Those of us with rare diseases are all definitely unique, but one fact came across loud and clear: Together, we are a very strong and a very vocal community. 1 in 10 Americans is inflicted with a rare disease and the time to accelerate progress is now! We are all fighting the battle of time and that is why this legislative advocacy is so critical.
I want to thank the Amyloidosis Research Consortium from the bottom of my heart for inviting me to attend as an amyloidosis advocate for those inflicted with amyloidosis. This was as much of a learning experience for me as it was for Congress.
The Amyloidosis Research Consortium is on the right path, working to identify treatments for amyloidosis (in many of its forms). In fact, their work with the FDA was referenced in the FDA presentation on Monday as being the standard they would like all other organizations to pursue for the FDA's programs to support clinical trials and their evaluation of novel therapies. The Amyloidosis Research Consortium is on the leading edge of providing guidelines to the FDA, establishing a path that other rare disease advocacy groups may follow for advancing the most promising and effective research.
All of us that are touched by amyloidosis are blessed to have our amyloidosis groups, researchers and doctors advocating and fighting for a cure for these deadly rare diseases. We live in an exciting time of science where amyloidosis will have focus and more treatments will become available.
Thank you all for allowing me to represent amyloidosis during Rare Disease Week on Capitol Hill.