Rare Disease Week

Rare Disease Week on Capitol Hill brings together rare disease community members from across the country to be educated on federal legislative issues, meet other advocates, and share their unique stories with legislators.

Read the recap from 2024 Rare Disease Week on Capitol Hill below!

Learn More

TAKE ACTION

Click the megaphone icon to access tools to advocate for important initiatives in the Rare Disease Community:

Call your Representative
Send a letter
Use your voice on social media

Advocacy Tips from a Batten Father

Advocacy Tips from Batten Mothers

Rare Disease Week 2024 Recap

For Rare Disease Week 2024, we asked our Batten community to lift their voices utilizing our advocacy module and submitting a pre-written letter to their legislator(s) urging them to elevate our mission as priority for funding and support. Below are the results:

Click here for a recap of 2024 Rare Disease Week on Capitol Hill by the Everylife Foundation.

BDSRA President & CEO Amy Fenton Parker attended Rare Disease Week on Capitol Hill in 2024 and met with staffers representing Ohio legislators. Click here to read the Q&A summary about her experience.

BDSRA Database Manager and rare disease sibling Noah Siedman gives updates on the Rare Disease Day at FDA virtual event and his meeting with BioMarin.

Rare Disease Week 2023 Recap

BDSRA was represented at the 2023 Rare Disease Week on Capitol event in Washington D.C. The event included a Rare Disease Day at NIH event, the Legislative Conference, Rare Disease Caucus, and meetings with State and Local Representatives to discuss issues concerning the rare disease community.

Click here for a recap of 2023 Rare Disease Week on Capitol Hill by the Everylife Foundation.

Watch the video below for takeaways from BDSRA staff and a board member regarding Rare Disease Day events.

BDSRA Marketing & PR Coordinator Patrick Kotnik met with two staffers representing Ohio legislators. Watch his summary below!

COLUMN: Rare Disease Week offers a storybook ending and road for optimism

We were witnesses to a storybook ending on a day dedicated to raising awareness and educating others on Rare Disease Day.

Ron Bartek, the Co-Founder and President of Friedreich’s Ataxia Research Alliance (FARA), received word shortly before Rare Disease Day at NIH wrapped up that the FDA approved the first-ever drug for Friedreich’s Ataxia (a rare inherited disease that causes progressive damage to your nervous system and movement problems) called SKYCLARYS for the treatment of people 16 years or older.

Relief, emotion, and catharsis consumed Bartek, who lost his son, Keith, at age 24 due to heart failure from Friedreich’s Ataxia.

Click here to read more

Interested in Advocating?

Feel free to share this BDSRA one-pager in your meetings with state and local representatives!

Get Involved

We encourage each family, friend, or advocate to learn more about how to advocate at the national and international levels. To learn more about how to advocate for topics such as newborn screening, early Medicaid access, and disability rights, check out the rest of our Advocacy Toolkit, or email Patrick Kotnik at patrick@bdsrafoundation.org.

The Burden of Rare Diseases

Every Life Foundation recently published a study on the burden of rare disease. The findings demand attention from researchers, policy makers, healthcare providers, employers. There is an urgent need to fund research, enhance awareness, and improve access to diagnosis, care, and treatment of rare disease. Contact your elected representatives, share the Study findings, and urge Congress to support important rare disease appropriations priorities that would advance critical rare disease research and therapy development at the National Institutes of Health, the Centers for Disease Control and Prevention, and the Food and Drug Administration.

Read The Full Report