2023 Rare Disease Week Recap
BDSRA was represented at the 2023 Rare Disease Week on Capitol Hill 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.
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.
