Leigh Syndrome Pipeline

Jun 04, 2025

Mitochondrial diseases, affecting an estimated 1.5 million individuals globally (approximately 1 in 5,000), represent a complex group of rare genetic disorders driven by mitochondrial dysfunction, disrupting cellular energy production. These conditions stem from mutations in mitochondrial DNA (mtDNA) or nuclear DNA...

May 26, 2025

Leigh syndrome, also called subacute necrotizing encephalomyelopathy, is a rare and serious genetic neurometabolic condition classified as a primary mitochondrial disease. It is associated with mutations in more than 75 genes, most of which are involved in cellular energy production. However, because many cases do ...