Despite advances in newborn screening and treatment of fatty acid oxidation disorders, patients with very-long-chain acyl-CoA dehydrogenase deficiency (VLCADD) continue to suffer from heart failure. Marie Norris will discuss her preliminary data which suggests that lipotoxicity, largely mediated by the accumulation of ceramides, is a major contributor to VLCADD-induced heart failure. Ms. Norris will discuss the role of ceramides, while addressing the mounting evidence that elevated ceramides contribute to heart failure in humans/rodents and that cardiac function improves with ceramide depletion.
“Understanding the New Recommendations on the Safety of Drug Use in Patients with a Primary Mitochondrial Disease” Clinical guidance is often sought when prescribing...
uy Miller, BioElectron’s CEO, and Matthew Klein, its Chief Medical Officer, will discuss the following: Challenges in the development of drugs for mitochondrial disease...
