The first disease-modifying treatment for spinal muscular atrophy (SMA), nusinersen, was approved by the US Food and Drug Administration (FDA) on Dec 23, 2016, and by the European Medicines Agency (EMA) on May 30, 2017. The approval was based on evidence of clinically meaningful improvements in motor milestones in young children with varying degrees of disease severity from two clinical trials (ENDEAR, NCT02193074, and CHERISH, NCT02292537). The regulatory approval is a historic development, but it is unlikely that the drug will be available to all patients who would benefit from treatment, unless its manufacturer offers a fairer price than the current cost of this drug.

中文翻译：

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治疗罕见疾病：以不公平的价格采取行动的时间

脊柱肌萎缩症（SMA）的首个疾病缓解疗法nusinersen已于2016年12月23日获得美国食品和药物管理局（FDA）的批准，并于2017年5月30日获得了欧洲药品管理局（EMA）的批准。批准的依据是两项临床试验（ENDEAR，NCT02193074和CHERISH，NCT02292537）中具有不同程度疾病严重程度的幼儿运动里程碑临床意义上的改善的证据。监管部门的批准是历史性的发展，但是除非该药物的制造商提供的价格比当前药物的价格更为合理，否则该药物不太可能向所有受益于治疗的患者使用。