尽管目前全球仅有5000名婴幼儿使用该药,但罕见病药物的高定价模式,叠加长期持续给药的治疗需求,让Vosoritide的商业表现持续走高。2025年,该药销售额达9.27亿美元,同比增长26%,是BioMarin的核心商业化增长引擎。
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She initially instructed her team to keep her in the dark about the nominations "to manage my expectations".
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"The whole audience were joining in - there is a group of ladies in their 80s who come every year and I saw them all punching the air along with everyone else.