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作为最常见的侏儒症类型,软骨发育不全(ACH)过去很长时间无特效疗法,直到2021年,“孤儿药之王”BioMarin研发的Vosoritide,作为首个获批药物,才开启了药物对症治疗时代。凭借独家优势,其2025年销售额高达9.27亿美元。

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