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

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

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 ...

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.

Bay Area family affected by Barth Syndrome frustrated by delay of approved treatments With the FDA recently rejecting a drug application that aims to help those living with Barth Syndrome, one Bay ...

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.

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.