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A 12-year-old boy is the first commercial patient in the world to receive an FDA-approved gene therapy for sickle cell disease, The New York Times reported May 6.Kendric Cromer is a 12-year-old boy from the suburbs of Washington, D.C., and the first to receive Lyfgenia, a gene therapy treatment created by Somerville, Mass.-based Bluebird Bio.
据《纽约时报》5月6日报道,一名12岁男孩是世界上第一位接受FDA批准的镰状细胞病基因治疗的商业患者。肯德里克·克罗默(KendricCromer)是一名来自华盛顿特区郊区的12岁男孩,也是第一位接受Lyfgenia治疗的男孩,Lyfgenia是由总部位于马萨诸塞州萨默维尔的蓝鸟生物公司(Bluebird Bio)开发的一种基因治疗方法。
Kendric's treatment, which costs about $3.1 million, is covered by his family's insurance. He underwent the first part of treatment at Washington, D.C.-based Children's National Hospital, in which physicians removed his bone marrow stem cells, which Bluebird will genetically modify for his treatment.
肯德里克的治疗费用约为310万美元,由其家人购买保险。他在华盛顿特区的儿童国家医院接受了第一部分治疗,医生移除了他的骨髓干细胞,蓝鸟将对其进行基因改造以进行治疗。
The modified cells will be returned in three months.The FDA gave two companies authorization to sell gene therapy to people with sickle cell disease, a genetic disorder that affects roughly 100,000 people, most of them Black. Bluebird estimates it can only treat 85 to 105 patients each year with sickle cell or beta thalassemia, who can receive a similar gene therapy.
修改后的细胞将在三个月内返回。FDA授权两家公司向镰状细胞病患者销售基因治疗,镰状细胞病是一种影响大约10万人的遗传疾病,其中大多数是黑人。蓝鸟估计,它每年只能治疗85至105名镰状细胞或β地中海贫血患者,这些患者可以接受类似的基因治疗。
Children's National, meanwhile, said it can accept only 10 gene therapy patients a year..
与此同时,国家儿童基金会表示,它每年只能接受10名基因治疗患者。。
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