The FDA has granted approval for Stealth Therapeutics' therapy targeting Barth syndrome, marking the first authorized mitochondria-targeted treatment. Barth syndrome is a rare genetic disorder characterized by impaired mitochondrial function leading to muscle weakness and cardiomyopathy. This regulatory greenlight represents a significant milestone, offering the first therapeutic option specifically designed to address the mitochondrial dysfunction underlying this condition. The approval by the FDA underscores the growing focus on precision medicines targeting subcellular organelles and expands treatment possibilities for patients with this rare disease.