Researchers at Tohoku University have discovered that an oral drug called MA-5 can improve both heart and muscle problems in Barth syndrome, a rare genetic disorder affecting 1 in 300,000 births ...

The Barth Syndrome Foundation (BSF), the only patient advocacy organization solely dedicated to saving lives through education, advances in treatments, and f ...

Developing a therapy for an ultra-rare condition has its challenges, including finding enough patients for clinical enrollment and convincing regulatory authorities that limited data prove the ...

An oral drug called MA-5 can improve both heart and muscle problems in Barth syndrome. Testing the drug on cells from Barth syndrome patients and in fruit fly models of the disease, the drug boosted ...

SAN FRANCISCO (KPIX) -- With the FDA recently rejecting a drug application that aims to help those living with Barth Syndrome, one Bay Area family is frustrated by the delay of approved treatments ...

Barth syndrome, a progressive, lethal, pediatric cardioskeletal disease often diagnosed at birth and with no approved therapies, affects ~150 individuals in the U.S. NEEDHAM, Mass., May 29, 2025 ...

Approximately 150 people live with Barth Syndrome nationwide, an ultra-rare genetic disorder that affects only males and impacts muscle and heart muscle health.

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