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Five of the world’s leading mitochondrial disease (mito) patient advocacy groups have announced they will together pledge USD$1 million to form and fund a patient-driven research network to fast-track treatments and potential therapies for Leigh syndrome. The Leigh Syndrome International Consortium will focus on improving diagnosis, therapeutic development and optimized patient care.

Leigh syndrome, a form of mitochondrial disease (mito), is a debilitating and potentially fatal genetic disorder that robs the body’s cells of the energy they need to function properly. 

The groups forming and funding this research network include; the Mito Foundation (Australia), the United Mitochondrial Disease Foundation (UMDF) and the People Against Leigh Syndrome (PALS) in the US, along with the UK’s Lily Foundation and Mitocon of Italy. 

Mito Foundation CEO, Sean Murray, says by working together the groups are accelerating potential treatments for Leigh syndrome patients who have significant unmet medical needs. 

“Leigh syndrome is an inherited neurometabolic disorder that affects the central nervous system,” Mr. Murray said. “This progressive disorder begins in infants between the ages of three months and two years, however the prognosis for those with the disease remains poor. “This innovative, multi-year project will fund vital research to help to improve diagnosis and establish global best practices of care for Leigh’s patients,” he said.

Brian T. Harman, President and CEO of UMDF, said by uniting together this network will drive more connections to help develop future treatments for Leigh syndrome patients. “No single organisation can take on a project of this scale alone. This collaboration between mitochondrial disease patient advocacy partners will help us leverage our existing connections to the patient community as well as elite international researchers,” Mr. Harman said.

Dr. Bruce M. Cohen, MD, FAAN, Pediatric Neurologist and Director of the NeuroDevelopmental Science Center at Akron Children’s Hospital, will lead the international scientific steering committee for the Leigh Syndrome International Consortium. “Initially, the network will focus on natural history data collection, outcome measure development and validation as well as pre-clinical data coordination,” Dr. Cohen said.

“Later this year, the steering committee plans to develop a “Request for Proposals” to support Leigh syndrome-focused grants.”

For more information about the Leigh Syndrome International Consortium visit www.leighsyndrome.org

 

 

© 2019 Int. Mito Patients