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沃姆斯利此前表示,公司是在回应FDA的“行政请求”,并强调“我们没有商业利益,没有科学研究,也没有在追求它”。FDA此次批准是基于对已发表病例报告和真实世界证据的系统审查,而非传统的临床试验。  据统计,脑叶酸缺乏症(FOLR1基因变异型)的发病率约为百万分之一。责任编辑:张俊 SF065

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