“Expanded Access” and “Compassionate Use” are terms that are utilized in our rare disease community, but what do they mean? How do patients learn about, get involved, better understand the qualifications, and talk with their doctor about participation in either of these programs? Join MitoAction and Dr. Koenig as we dive deep into better understanding the differences and overlap between “Expanded Access” and “Compassionate Use” therapies.
Join MitoAction and Matthew Klein and Francesco Bibbiani from PTC Therapeutics for our November Mito Expert Series presentation. About the Speakers Matthew B. Klein,...
Siblings of children with disabilities play important roles in their families, and often experience similar concerns and opportunities as parents. This session will present...
Dr. Fran Kendall of VMP Genetics discusses “Is it really Mito? When an alternative diagnosis should be considered.” Talking points include: Clinical red flags...
