Oral drug MA-5 can improve heart and muscle problems in Barth syndrome

Oral drug MA-5 can improve heart and muscle problems in Barth syndrome

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 worldwide with no current cure.

First oral drug shows promise for Barth syndrome heart and muscle symptoms

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 worldwide with no current cure.