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Leigh syndrome is a neurometabolic condition caused by pathogenic variants, or genetic differences, in one of more than 75 genes. These genes all play important roles in the creation of mitochondria, or the powerhouses of the cell. Mitochondria make the energy the body needs to function properly, including actions like lifting your arms or inflating your lungs with each breath. People with Leigh syndrome have mitochondria that do not work properly, leading to a progressive loss of mental and movement abilities and eventually, vision, kidney, heart and respiratory complications. Leigh syndrome typically presents during infancy or early childhood and can be fatal. There is currently no targeted treatment for Leigh syndrome, but supportive treatment can help to improve some symptoms.
The latest on our research
We are jumpstarting our Leigh syndrome program to accelerate research on this condition and to ultimately improve treatment options. We welcome all patients and caregivers interested in participating and spreading the word to the Leigh syndrome community.