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 ...

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.

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.

Elamipretide is being developed for Barth syndrome, an inherited mitochondrial disorder that causes an enlarged and weakened heart, skeletal muscle problems and infections and affects around 150 ...

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, ...