Rare Disease Day on Capitol Hill

Rare Disease Day on Capitol Hill

Today is #RareDiseaseDay – a day for advocating, not really celebrating IMO. Let’s celebrate our differences by who we are as individuals, not by our diagnosis.

Achondroplasia is caused by a genetic mutation of the FGFR3 gene. Times are changing and it’s important to be a voice that supports the science behind it because it’s way more than just being short. Currently, there are four pharmaceutical companies developing treatments for achondroplasia.

We need more FDA Rare Disease Orphan Drug research funding.  Without it, research and development slow down making the waiting game longer for anyone with a rare disease.

This week, I met with fellow Arkansan, U.S. Congressman French Hill, and asked him to consider joining the Rare Disease Caucus and to support more funding for an FDA Rare Disease Center of Excellence, as well as a Natural History Grants Program and Orphan Products Clinical Trial Grants. I’ll be watching his performance and votes and hope you will too. 💜


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