Primary Mitochondrial Diseases
A Rare Disease Virtual Workshop
May 22, 2025 | 10am-3:30pm (eastern)
The Reagan-Udall Foundation for the FDA , in collaboration with the Food and Drug Administration (FDA), hosted a virtual, public workshop on "Primary Mitochondrial Diseases."
As part of the Foundation's rare disease focus, this workshop explored opportunities to optimize therapeutic development addressing primary mitochondrial diseases and best practices in designing and interpreting clinical studies that can produce meaningful data to accelerate drug development. Speakers included patient advocates, academic researchers, regulated industry, healthcare providers, and other key stakeholders.