Carlos T. Moraes, PhD, professor of Neurology at the University of Miami’s Miller School of Medicine in Florida, has an ongoing MDA research grant (totaling $300,000 over three years) to develop gene-editing enzymes that can be used to correct genetic mutations causing mitochondrial myopathies. Mitochondrial diseases caused by mutations in the mitochondrial DNA (mtDNA) are most . . .
Filmmaker Tom Caamano, who received a surprise diagnosis of mitochondrial disease in 2009, perseveres despite symptoms, which include muscle weakness and fatigue, that threatened to derail his career. Today, he is not only thriving, he’s helping others facing similar diagnoses feel empowered, too.
For space anthropologist Keirsten Snover, living unlimited means going where no man or woman has gone before. “As far back as I can remember, I always loved science,” Keirsten says. “I have always been fascinated by all the different fields.” Keirsten has mitochondrial myopathy. As a result of the disease’s progression, she uses a wheelchair . . .
When Jayden Long learned four years ago that the firefighters in his hometown of Nampa, Idaho, had to work on Christmas, he devised a plan. “He wanted to deliver baked goods to the firefighters on Christmas day,” recalled his mother, Shellie. “So we baked just about all night and delivered trays of food to three . . .
Since its inception, MDA has invested more than $1 billion in neuromuscular disease research to uncover new treatments and cures. In 2016 alone, MDA awarded 66 new research, development and research infrastructure grants with a total funding commitment of more than $17 million. These and other MDA grants fund research projects designed to uncover the . . .
This summer MDA challenged our families, friends, sponsors, staff and others around the nation to share their stories about living life without limits despite the challenges of neuromuscular disease. Some shared moments tied to pursuing an education or career that was thought to be out of reach. Some talked about skydiving, hiking or running . . .
Stealth Bio Therapeutics has reported encouraging results from a phase 2 clinical trial to evaluate elamipretide for the treatment of muscle weakness caused by mitochondrial disease. Elamipretide is an experimental drug designed to modify disease by helping to restore normal energy production in mitochondria and decrease oxidative stress. In the completed MMPOWER trial, investigators treated . . .