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诺华的天价基因疗法Zolgensma，在欧洲获批治疗脊柱性肌肉萎缩症

2020-05-20 MedSci原创 MedSci原创

基因疗法Zolgensma脊柱性肌肉萎缩症
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一年前，Zolgensma在美国获得批准，成为世界上最昂贵的药物，每位患者的价格为210万美元。

诺华公司的基因疗法Zolgensma（onasemnogene abeparvovec）获得了欧洲EMA有条件的批准，用于治疗1型脊柱性肌肉萎缩症（SMA）（疾病最严重形式）患者，以及具有三个SMN2基因拷贝的SMA患者。

一年前，Zolgensma在美国获得批准，成为世界上最昂贵的药物，每位患者的价格为210万美元，三月份在日本也获批，价格被下调至155万美元。

去年该药物的销售额达到3.61亿美元，在今年第一季度又增加了1.70亿美元。GlobalData预测Zolgensma最高销售额将达到每年25亿美元。

在欧洲，每年约有550-600名婴儿患有SMA，会导致运动神经元快速且不可逆转的丧失，影响肌肉功能并引起呼吸、吞咽和运动问题。SMA是一种毁灭性疾病，在最严重的情况下，婴儿的预期寿命仅为两年多一点。

Zolgensma令人兴奋的地方在于它作为SMA的一次性疗法，随着时间的推移，既可以改善SMA患者的临床疗效，又可以降低整体护理费用。

原始出处：

http://www.pmlive.com/pharma_news/novartis_claims_eu_approval_for_sma_gene_therapy_zolgensma_1340543

评论区 (9)
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    #基因疗法Zolgensma#

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    2020-06-18 12498a1cm74暂无昵称

    #Zolgensma#

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    2020-08-02 chendoc252

    #萎缩#

    0

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style='color:#2F92EE;'>#肌肉萎缩症#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=82, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81019, encryptionId=b05281019f8, topicName=肌肉萎缩症)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c28719479710, createdName=12498768m19(暂无昵称), createdTime=Fri May 22 01:59:28 CST 2020, time=2020-05-22, status=1, ipAttribution=)]
    2020-05-22 nian.wang1982

    #诺华#

    0

  5. 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style='color:#2F92EE;'>#肌肉萎缩症#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=82, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81019, encryptionId=b05281019f8, topicName=肌肉萎缩症)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c28719479710, createdName=12498768m19(暂无昵称), createdTime=Fri May 22 01:59:28 CST 2020, time=2020-05-22, status=1, ipAttribution=)]
    2020-05-22 ms9474365431325850

    #欧洲#

    0

  6. 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ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1388934, encodeId=44ed1388934d8, content=<a href='/topic/show?id=a75a1642585' target=_blank style='color:#2F92EE;'>#SMA#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=59, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=16425, encryptionId=a75a1642585, topicName=SMA)], attachment=null, authenticateStatus=null, createdAvatar=http://thirdwx.qlogo.cn/mmopen/vi_32/Q0j4TwGTfTK17mLkWpia3T92NlEWTkEhrhZa7XPNic2JqQumEicZXGYFG6Ww5SCIl0Uq62bhhPnibAqIZDHtRva92A/132, createdBy=e0752500025, createdName=江川靖瑶, createdTime=Fri May 22 01:59:28 CST 2020, time=2020-05-22, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1539233, encodeId=4de215392333d, content=<a href='/topic/show?id=6505108534a' target=_blank style='color:#2F92EE;'>#lgE#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=77, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=10853, encryptionId=6505108534a, topicName=lgE)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=beb613117181, createdName=HNYYM, createdTime=Fri May 22 01:59:28 CST 2020, time=2020-05-22, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1584185, encodeId=c4a7158418555, content=<a href='/topic/show?id=f3308101eee' target=_blank style='color:#2F92EE;'>#肌肉萎缩#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=85, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81017, encryptionId=f3308101eee, topicName=肌肉萎缩)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=177b17012429, createdName=12498ebem24暂无昵称, createdTime=Fri May 22 01:59:28 CST 2020, time=2020-05-22, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1614086, encodeId=12ee161408658, content=<a href='/topic/show?id=b05281019f8' target=_blank style='color:#2F92EE;'>#肌肉萎缩症#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=82, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81019, encryptionId=b05281019f8, topicName=肌肉萎缩症)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c28719479710, createdName=12498768m19(暂无昵称), createdTime=Fri May 22 01:59:28 CST 2020, time=2020-05-22, status=1, ipAttribution=)]
    2020-05-22 江川靖瑶

    #SMA#

    0

  7. 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style='color:#2F92EE;'>#肌肉萎缩症#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=82, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81019, encryptionId=b05281019f8, topicName=肌肉萎缩症)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c28719479710, createdName=12498768m19(暂无昵称), createdTime=Fri May 22 01:59:28 CST 2020, time=2020-05-22, status=1, ipAttribution=)]
    2020-05-22 HNYYM

    #lgE#

    0

  8. 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    2020-05-22 12498ebem24暂无昵称

    #肌肉萎缩#

    0

  9. 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style='color:#2F92EE;'>#肌肉萎缩症#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=82, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81019, encryptionId=b05281019f8, topicName=肌肉萎缩症)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c28719479710, createdName=12498768m19(暂无昵称), createdTime=Fri May 22 01:59:28 CST 2020, time=2020-05-22, status=1, ipAttribution=)]
    2020-05-22 12498768m19(暂无昵称)

    #肌肉萎缩症#

    0

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