Join MitoAction CEO, Kira Mann, Barth Syndrome Foundation CEO, Emily Milligan, and Stealth BioTherapeutics CEO, Reenie McCarthy for a discussion as we navigate the FDA's most recent update on the application for elamipretide, a crucial treatment for the Barth syndrome community.
When a genetic variant is shared by only a handful of individuals worldwide, what does it mean for diagnosis, treatment, and research? In this session, we’ll explore the complexities of interpreting ultra-rare genetic mutations, especially in the context of mitochondrial disease. How do clinicians and geneticists determine whether a novel or rare variant is pathogenic? What frameworks are used […]