There are two new MCADD trials that have started in recent months. One explores the use Dojolvi (triheptanoin) and the …
Expert Series: Ultra-Rare Mitochondrial Diseases: Development Challenges and Opportunities
Join CEO of Stealth Biotherapeutics Reenie McCarthy, and special guest Shelley Bowen, Director of Family Services and …
Expert Series: Modeling mitochondrial diseases with patient-derived stem cells and brain organoids
In this talk, Dr. Alessandro Prigione will discuss how stem cells are generated from patients to derive models of the …
Expert Series: Ceramides: The Unmasked Drivers of VLCADD-Induced Heart Failure
Despite advances in newborn screening and treatment of fatty acid oxidation disorders, patients with very-long-chain …
Expert Series: LCHADD Retinopathy Update: Moving Toward a Treatment
Having a better understanding of what things are associated with LCHADD retinopathy can provide insight towards …
