The Giants in Mitochondrial Diseases Work Together for Leigh Syndrome

As originally reported in Scimex from a press release by The MITO Foundation, the main five patient advocacy groups on mitochondrial diseases have pledged to work together to afford 1 million USD for Leigh Syndrome. They will create a patient driven research network with hopes of finding new treatments and therapies for the debilitating genetic condition that affects the central nervous system.

What is Leigh syndrome? 

Leigh syndrome is an inherited severe neurological disorder which begins in infancy and is progressive, worsening over time. It is a neurometabolic disease which sucks the energy from the body’s cells so that they cannot function as they should. This causes symptoms such as throwing up, diarrhea, trouble with swallowing, difficulty eating, weight gain, stunted growth, problems with muscles and balance, and more. It is potentially fatal, with most patients passing away within the first three years.

The current project 

This project brings together the giants in their overarching focus, mitochondrial diseases, in order to create a push for a disease that is under researched and lacks medical attention. It will be a multi-year project with an innovative approach. By bringing together big players that together have the tools, money, and influence to make tangible change. The CEO’s of the various organizations believe this will spur the progress that’s needed, but still unmet. Brian T. Harman, President and CEO of U.S. United Mitochondrial Disease Foundation (UMDF) believes the coalition of the big organizations will build connections and networks that will then share findings and information as well as create a global standard practice for the most effect treatment options. He believes how the magnitude of the project is necessary to tackle the weight of the issue. Harman described, ““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.”
The leaders are creating an action plan, beginning with data collection, standardized measurements, and pre-clinical coordination, before moving on to proposing more focused grants and specific research tasks. With collaboration and sharing information, they’re bound to find more than ever before.

What are your thoughts on advocacy groups collaborating more frequently? Share your stories, thoughts, and hopes with the Patient Worthy community!

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