Media Release
From: Mito FoundationLeigh 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 U.S United Mitochondrial Disease Foundation (UMDF) and the People Against Leigh Syndrome (PALS), along with The UK’s Lily Foundation and the 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 & CEO of UMDF, said by uniting together this network will drive more connections to help develop future treatments for Leigh Syndrome patients.
“No single organization 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
Media contact: Sean Murray 0422 819 975
ABOUT MITOCHONDRIAL DISEASE
Mitochondrial disease (mito) is a debilitating genetic disorder that robs the body’s cells of energy, causing multiple organ dysfunction or failure and sometimes death. When mitochondria are faulty, the body does not get the correct level of energy it needs to function.
One in 200 people, or more than 120,000 Australians, may carry genetic changes that put them at risk of developing mito. One Australian child born each week will develop a severely disabling or life-threatening form of mito.There are many forms of mito; it is highly complex and can cause any symptom in any organ at any age. Mito is terminal; there are no cures and few effective treatments.
ABOUT THE FOUNDING ADVOCACY GROUPS
The Mito Foundation supports people affected by mitochondrial disease, funds essential research into the prevention, diagnosis, treatment, and cures of mitochondrial disorders, and increases awareness and education. In its ten years of operation, the foundation has raised more than $17 million which has been invested in research and community support. This puts the Mito Foundation in the top five percent by revenue of the 60,000 registered charities in Australia and the top few percent funding medical research.
The United Mitochondrial Disease Foundation (UMDF) promotes research and education for the diagnosis, treatment and cure of mitochondrial diseases and to provide support for affected individuals and families. Since its inception, the UMDF has funded more than $12 million in research, making it the leading non-governmental contributor of grants focused solely on mitochondrial disease. People Against Leigh Syndrome (PALS) was formed in 2013 after William Martin, son of Neil and Lori Martin, was diagnosed with Leigh syndrome. PALS has built awareness of this unknown disease and raised funds to support the medical community in their quest to develop viable treatments and ultimately a cure. The Lily Foundation is committed to finding a cure for mitochondrial disease through funding research, raising awareness and family support. Mitocon was formed in 2007 by a group of parents of children suffering from mitochondrial disease. Over the years, Mitocon has expanded and has become the leading association in Italy for all mitochondrial patients, adults and children, and for their families and the entire scientific community.
