美国食品和药物管理局批准了第一个针对12岁及以上儿童罕见血病的治疗. FDA approves first targeted treatment for rare pediatric blood disorder in patients 12 and older.

美国食品和药物管理局批准了为12岁及以上儿童治疗罕见的血液疾病的首个治疗方法, The FDA has approved the first treatment for a rare blood disorder in pediatric patients aged 12 and older, marking a significant advancement in care for young people with the condition. 批准的依据是临床试验结果,表明治疗有效减少了症状并改善了结果。 The approval is based on clinical trial results showing the therapy effectively reduces symptoms and improves outcomes. 治疗对象为患有与疾病相关的特定基因突变的患者,在几乎没有替代品的情况下提供有针对性的选择。 The treatment is intended for patients with a specific genetic mutation linked to the disorder, offering a targeted option where few alternatives existed. 这项决定旨在解决这些弱势人口未得到满足的医疗需求。 This decision aims to address unmet medical needs in this vulnerable population.