The FDA has granted long-awaited approval to Stealth Therapeutics' novel treatment for Barth syndrome, marking the first mitochondria-targeted therapy available for this rare genetic disorder. Barth syndrome is characterized by multi-system dysfunction including cardiomyopathy and muscle weakness, with limited treatment options to date. Approval of this therapy represents a milestone in addressing mitochondrial pathology directly, offering a new clinical avenue for affected patients. This development, reported by BioCentury and dated Sept. 19, 2025, underscores the growing focus on targeted interventions for mitochondrial diseases.