The FDA granted long-anticipated approval to Stealth Therapeutics’ Barth syndrome therapy, marking the first mitochondria-targeted treatment authorized for this rare genetic disorder. Barth syndrome causes debilitating cardiac and muscular impairments, and this regulatory milestone provides a novel treatment pathway for affected patients. The approval is expected to enhance therapeutic options and catalyze further development of mitochondria-focused interventions. Stealth’s accomplishment underscores the evolving frontier in rare disease drugs.