Researchers at Tohoku University have discovered that an oral drug called MA-5 can improve both heart and muscle problems in ...

With the FDA recently rejecting a drug application that aims to help those living with Barth Syndrome, one Bay Area family is ...

With the FDA recently rejecting a drug application that aims to help those living with Barth Syndrome, one Bay Area family is ...

Gilbert has Barth syndrome, a rare, genetic, and often fatal disease that weakens the heart and immune system. About 150 people in the U.S. are known to have it.

The FDA denied a lifesaving drug for Barth syndrome. Now, one Colorado family fears their baby’s only treatment could be pulled.

Following five years of discussions and a positive advisory committee recommendation, FDA proposes a potential accelerated approval pathway requiring resubmission of the elamipretide NDA Barth ...

BOSTON, May 29, 2025--The Barth Syndrome Foundation (BSF) today responded to new developments from the U.S. Food and Drug Administration (FDA) regarding elamipretide, a potential treatment for the ...

Barth syndrome, a progressive, lethal, pediatric cardioskeletal disease often diagnosed at birth and with no approved therapies, affects ~150 individuals in the U.S.