Leber's hereditary optic neuropathy (LHON) is a mitochondrial disease characterized by central vision loss and a poor visual prognosis. Marcela Votruba discusses new insights and approaches to therapy. Professor Marcela Votruba is a clinically qualified and clinically active Professor of Ophthalmology at Cardiff University School of Optometry & Vision Sciences and an Honorary Consultant Ophthalmologist at the University Hospital of Wales, Cardiff.