Barth syndrome is a rare genetic disorder that mostly affects males and can cause problems with the heart, muscles, and immune system. Common symptoms of Barth syndrome include weak muscles, tiredness ...

In a Nature Metabolism paper published today, researchers from the University of Pittsburgh detail a potential new target and a small-molecule drug candidate for treating Barth syndrome, a rare, ...

Gilbert Dryden probably only has enough medication to get him through the end of October, his mother, Madison, figures. Seven-month-old Gilbert has a rare genetic condition called Barth syndrome, one ...

Boys suffering from Barth syndrome, a genetic determined disease with a very high mortality rate, finally have hope for a cure. What has contributed to this finding is research of Dr. habil Karolina ...

ORLAND PARK, Ill. (WLS) -- A baby boy in the Chicago area is battling a rare disease and desperately needs a drug treatment for a better life. The drug has not been FDA approved, and it could ...

FDA advisors indicated a possible path forward for elamipretide as a treatment for Barth syndrome despite the lack of definitive evidence. On Thursday, the Cardiovascular and Renal Drugs Advisory ...

AURORA, Colo. — Five-month-old Gilbert Dryden is thriving—cooing, smiling and gaining weight after spending the first 80 days of his life in the hospital. But his parents said his progress, and ...

MA-5 treatment comprehensively improves Barth syndrome pathology. In Barth syndrome models (left panel, A.), iPS cell-derived myoblasts show decreased mitochondrial function and increased endoplasmic ...

Another ultra-rare therapy will soon be subject to the gaze of an FDA advisory committee, with the fate of more than 200 boys diagnosed with Barth syndrome worldwide in the balance. On Thursday, the ...