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 ...

Barth Syndrome: A rare X‐linked genetic disorder characterised by cardiomyopathy, skeletal myopathy, growth delay and metabolic abnormalities due to mutations in the TAZ gene.

Barth syndrome is a rare genetic disorder with no known cure. Researchers at Tohoku University examined a new oral drug called MA-5 that could provide life-changing relief to these young patients.

It's the only drug for Barth Syndrome clinical trials. After several delays, however, the FDA announced it will not be approving the drug application.

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

By Sooji Nam. Click here for updates on this story. SAN FRANCISCO — With the FDA recently rejecting a drug application that aims to help those living with Barth Syndrome, one Bay Area family is ...