林业发展局根据试验中肌肉功能的改善,批准巴思综合症Forzinity for Barth Instate,这是首次治疗稀有遗传疾病。 FDA approves Forzinity for Barth syndrome, the first treatment for the rare genetic disorder, based on improved muscle function in trials.

美国食品和药物管理局已对Forzinity (elamipretide) 给予加速批准,用于治疗巴斯综合征,这是一种罕见的遗传疾病,主要影响男性,标志着这种疾病的首次治疗. The FDA has granted accelerated approval to Forzinity (elamipretide) for Barth syndrome, a rare genetic disorder affecting primarily males, marking the first treatment for the condition. 经2025年9月19日批准,该药物改善了线粒体功能,并以临床试验结果为依据,显示肌肉力和体能增强。 Approved on September 19, 2025, the drug improves mitochondrial function and is based on clinical trial results showing enhanced muscle strength and physical performance. 目前批准为年龄在12岁及以上、体重至少为66磅的病人提供这项服务,并不断努力扩大年轻人获得治疗的机会。 It is currently approved for patients aged 12 and older weighing at least 66 pounds, with ongoing efforts to expand access to younger individuals. 批准是在多年的拖延和家庭宣传之后进行的,需要经过批准后的审判才能确认长期福利。 The approval follows years of delays and advocacy from families, and requires a post-approval trial to confirm long-term benefits. 常见副作用是轻微的注射点反应。 Common side effects are mild injection-site reactions.