What Is Leigh Syndrome
Leigh syndrome is a progressive neurometabolic disorder that can cause deterioration of the central nervous system, including the brain, spinal cord, and optic nerve. It has a general onset in infancy or childhood, often after a viral infection, but can also occur in teens and adults. It is characterised on magnetic resonance imaging (MRI) by visible necrotising (dead or dying tissue) lesions on the brain, particularly in the midbrain and brainstem. The prognosis for Leigh syndrome is poor. Depending on the defect, individuals typically live anywhere from a few years to the mid-teens.
The Leigh Syndrome Consortium
In an effort to move fast toward treatments and potential therapies for Leigh Syndrome, five of the world’s leading mitochondrial disease patient advocacy groups have joined together to form and fund the Leigh Syndrome Consortium. Two U.S. based organizations, the United Mitochondrial Disease Foundation (UMDF) and People Against Leigh Syndrome (PALS), along with Australian based Mito Foundation, The Lily Foundation of the U.K., and Mitocon of Italy, have pledged $1 million USD to support this innovative, multi-year project to fund research with a focus on improving diagnosis, therapeutic development and optimized patient care for Leigh’s patients.
The Leigh Syndrome Roadmap Project
The project is designed to fast-track potential treatments and cures for patients who have significant unmet medical needs. To do so, the project will pull together elite international researchers, many of whom already engage with one or more mitochondrial disease patient groups but not as a cohesive international network.