Researchers uncover a promising new therapeutic target for Barth syndrome
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The Hospital for Sick Children Sep 3 2025 Researchers at The Hospital for Sick Children (SickKids) have uncovered a promising new therapeutic target for Barth syndrome, a rare genetic condition with no current cure. Barth syndrome is an X-linked genetic condition affecting about 500 people worldwide, most of whom are males. The rare condition causes muscle weakness, frequent infections and cardiomyopathy . While heart transplants can manage cardiac symptoms, many children with Barth syndrome
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