Tohoku University Jul 1 2025 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. Barth syndrome is caused by mutations in the TAZ gene that leave patients—mostly young boys—with weakened hearts, muscle fatigue, and increased rates of infection. Many require heart transplants, and current treatments only manage symptoms