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

Barth Syndrome Foundation and our international affiliates comprise the only global network of families, healthcare providers, and researchers solely driven by the mission to save lives through ...

In human muscle cells derived from Barth syndrome iPS cell models, MA-5 corrected abnormal mitochondrial structures and reduced cellular stress markers. When tested in Drosophila with Barth syndrome, ...

An oral drug called MA-5 can improve both heart and muscle problems in Barth syndrome. Testing the drug on cells from Barth syndrome patients and in fruit fly models of the disease, the drug boosted ...

Barth Syndrome affects 130 known American men and boys. It occurs only in the male population and causes an enlarged heart and overall muscle weakness.

Developing a therapy for an ultra-rare condition has its challenges, including finding enough patients for clinical enrollment and convincing regulatory authorities that limited data prove the ...

Barth syndrome is a rare genetic disorder characterized by muscle weakness and heart abnormalities. The Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for ...

There are about 300 people worldwide living with Barth Syndrome, and 8-year-old Deacon Dague in Hollywood is one of them. His family hopes to raise more awareness so that there will be a cure one day.

Stealth Biotherapeutics Inc. had hoped the U.S. FDA would have approved its lead candidate, elamipretide, as the first treatment for Barth syndrome by now. Instead, it’s headed to a meet-up with the ...