FDA delays could end vital treatment for rare disease patients
The FDA is failing rare disease patients. I’m one of them.
I’ve been on elamipretide for three years now. It is the only treatment that has made a meaningful difference in my life with primary mitochondrial myopathy—a degenerative genetic condition that impairs how my cells convert food and oxygen into energy. Mitochondria provide over 90 percent of the energy our bodies need; when they fail, the highest-demand tissues—like muscle, brain, and …
