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FDA暂时搁置了对鞘内施用SMA基因疗法Zolgensma的研究

2019-10-30 Allan MedSci原创

SMAZolgensma基因疗法
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诺华制药近日宣布，FDA暂时搁置了在鞘内施用基因疗法Zolgensma（onasemnogene abeparvovec）（也称为AVXS-101）的试验。该公司指出，在临床前动物研究中，基因疗法Zolgensma有时会导致神经元细胞体变性或丧失。

诺华制药近日宣布，FDA暂时搁置了在鞘内施用基因疗法Zolgensma（onasemnogene abeparvovec）（也称为AVXS-101）的试验。该公司指出，在临床前动物研究中，基因疗法Zolgensma有时会导致神经元细胞体变性或丧失。

根据诺华公司的研究，目前正在I / II期试验中对年龄在5岁以下的2型脊髓性肌萎缩症（SMA）的患者进行鞘内施用Zolgensma。静脉注射疗法在今年早些时候获得了美国的批准，用于治疗两岁以下患有SMN1基因双等位基因突变的SMA患儿。该制药商指出，试验的部分搁置不会影响Zolgensma上市或正在进行的静脉临床研究。


原始出处：

http://www.firstwordpharma.com/node/1677375#axzz63qZhPxYq

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    2020-06-17 12498a1cm74暂无昵称

    #Zolgensma#

    0

  2. [GetPortalCommentsPageByObjectIdResponse(id=1640553, encodeId=ebb1164055391, content=<a href='/topic/show?id=af9d19083bc' target=_blank style='color:#2F92EE;'>#Zolgensma#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=51, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=19083, encryptionId=af9d19083bc, topicName=Zolgensma)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=bf0722658792, createdName=12498a1cm74暂无昵称, createdTime=Wed Jun 17 13:35:00 CST 2020, time=2020-06-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1688064, encodeId=08ad168806436, content=<a href='/topic/show?id=779441e9967' target=_blank style='color:#2F92EE;'>#基因疗法Zolgensma#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=45, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=41799, encryptionId=779441e9967, topicName=基因疗法Zolgensma)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=36fb28449261, createdName=124987c6m45暂无昵称, createdTime=Wed Mar 04 16:35:00 CST 2020, time=2020-03-04, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1388918, encodeId=9d0f138891834, content=<a href='/topic/show?id=a75a1642585' target=_blank style='color:#2F92EE;'>#SMA#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=45, 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 Nov 01 13:35:00 CST 2019, time=2019-11-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1539225, encodeId=1e1b153922515, content=<a href='/topic/show?id=6505108534a' target=_blank style='color:#2F92EE;'>#lgE#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=48, 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 Nov 01 13:35:00 CST 2019, time=2019-11-01, status=1, ipAttribution=)]
    2020-03-04 124987c6m45暂无昵称

    #基因疗法Zolgensma#

    0

  3. [GetPortalCommentsPageByObjectIdResponse(id=1640553, encodeId=ebb1164055391, content=<a href='/topic/show?id=af9d19083bc' target=_blank style='color:#2F92EE;'>#Zolgensma#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=51, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=19083, encryptionId=af9d19083bc, topicName=Zolgensma)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=bf0722658792, createdName=12498a1cm74暂无昵称, createdTime=Wed Jun 17 13:35:00 CST 2020, time=2020-06-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1688064, encodeId=08ad168806436, content=<a href='/topic/show?id=779441e9967' target=_blank style='color:#2F92EE;'>#基因疗法Zolgensma#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=45, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=41799, encryptionId=779441e9967, topicName=基因疗法Zolgensma)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=36fb28449261, createdName=124987c6m45暂无昵称, createdTime=Wed Mar 04 16:35:00 CST 2020, time=2020-03-04, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1388918, encodeId=9d0f138891834, content=<a href='/topic/show?id=a75a1642585' target=_blank style='color:#2F92EE;'>#SMA#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=45, 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 Nov 01 13:35:00 CST 2019, time=2019-11-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1539225, encodeId=1e1b153922515, content=<a href='/topic/show?id=6505108534a' target=_blank style='color:#2F92EE;'>#lgE#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=48, 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 Nov 01 13:35:00 CST 2019, time=2019-11-01, status=1, ipAttribution=)]
    2019-11-01 江川靖瑶

    #SMA#

    0

  4. [GetPortalCommentsPageByObjectIdResponse(id=1640553, encodeId=ebb1164055391, content=<a href='/topic/show?id=af9d19083bc' target=_blank style='color:#2F92EE;'>#Zolgensma#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=51, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=19083, encryptionId=af9d19083bc, topicName=Zolgensma)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=bf0722658792, createdName=12498a1cm74暂无昵称, createdTime=Wed Jun 17 13:35:00 CST 2020, time=2020-06-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1688064, encodeId=08ad168806436, content=<a href='/topic/show?id=779441e9967' target=_blank style='color:#2F92EE;'>#基因疗法Zolgensma#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=45, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=41799, encryptionId=779441e9967, topicName=基因疗法Zolgensma)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=36fb28449261, createdName=124987c6m45暂无昵称, createdTime=Wed Mar 04 16:35:00 CST 2020, time=2020-03-04, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1388918, encodeId=9d0f138891834, content=<a href='/topic/show?id=a75a1642585' target=_blank style='color:#2F92EE;'>#SMA#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=45, 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 Nov 01 13:35:00 CST 2019, time=2019-11-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1539225, encodeId=1e1b153922515, content=<a href='/topic/show?id=6505108534a' target=_blank style='color:#2F92EE;'>#lgE#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=48, 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 Nov 01 13:35:00 CST 2019, time=2019-11-01, status=1, ipAttribution=)]
    2019-11-01 HNYYM

    #lgE#

    0

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