Saturday, September 5, 2020

Broad Institute launches new effort to study rare neuromuscular disorder

Broad Institute, NEWS / 09.3.20. By Leah Eisenstadt.
A new research and drug discovery effort at the Broad Institute of MIT and Harvard is taking aim at the rare, inherited movement disorder Friedreich’s ataxia (FA), which causes progressive damage to the nervous system. FA arises from genetic mutations that lead to dysfunction of the cell’s energy-producing organelles called mitochondria.
“The goal of the Friedreich's Ataxia Accelerator is to nucleate a small group of investigators who will bring the power of genomics to this debilitating disease,” said Mootha, who is also a Howard Hughes Medical Institute investigator and professor of medicine at Harvard Medical School and Massachusetts General Hospital.