近日，一款“一针抵一套豪宅”的罕见病药物引发业内关注。该药物是诺华最新获得美国FDA批准用于治疗两岁及以上已确诊携带运动神经元存活基因1（SMN1）基因突变的脊髓性肌萎缩症（SMA）患者的基因疗法Itvisma。 随着越来越多高价基因疗法进入市场，这些单剂量、有望治愈疾病的治疗方式不断刷新着美国高价药物榜单。不过，高昂的价格、市场推广的挑战、医保报销等问题仍困扰基因药物的发展。 据诺华披露，...
Source Link近日，一款“一针抵一套豪宅”的罕见病药物引发业内关注。该药物是诺华最新获得美国FDA批准用于治疗两岁及以上已确诊携带运动神经元存活基因1（SMN1）基因突变的脊髓性肌萎缩症（SMA）患者的基因疗法Itvisma。 随着越来越多高价基因疗法进入市场，这些单剂量、有望治愈疾病的治疗方式不断刷新着美国高价药物榜单。不过，高昂的价格、市场推广的挑战、医保报销等问题仍困扰基因药物的发展。 据诺华披露，...
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