Mitochondrial Disease Workshop Shows US FDA Commitment To First Approvals

 
• By Kate Rawson

First FDA-hosted workshop on rare family of mitochondrial diseases offers plenty of opportunities to talk challenges in R&D – and identify some solutions.

Mitochondria (red) are organelles found in most cells. They generate a cell's chemical energy.
Mitochondrial disorders include Barth syndrome, mitochondrial cardiomyopathies, Leber’s hereditary optic neuropathy, Friedreich’s ataxia, Alpers syndrome and Leigh syndrome. • Source: NIH National Institute of Child Health and Human Development

More from United States

More from North America