英国NICE批准Alnylam的基因干扰药物Onpattro用于治疗hATTR淀粉样变性

2019-07-10 不详 MedSci原创

英国国家健康与护理卓越研究所(NICE)在2018年12月发布 "否"之后,重新建议使用Alnylam的Onpattro(patisiran)治疗遗传性转甲状腺素介导的淀粉样变性病(hATTR淀粉样变性)。

英国国家健康与护理卓越研究所(NICE)在2018年12月发布 "否"之后,重新建议使用Alnylam的Onpattro(patisiran)治疗遗传性转甲状腺素介导的淀粉样变性病(hATTR淀粉样变性)。

这种疾病是由一种叫做转甲状腺素蛋白(TTR)的异常沉积和积聚引起的,是一种进行性的可危及生命的疾病。Onpattro通过使用干扰RNA(RNAi)阻断大部分TTR蛋白的产生,减少损害全身神经和器官的TTR蛋白积聚从该疾病的根本原因上来进行解决。

NICE决定改变其对Onpattro的立场,使Alnylam重新回到hATTR治疗的尖端位置。

皇家自由医院国家淀粉样变性中心负责人菲利普霍金斯教授表示,这一决定标志着"在治疗hATTR淀粉样变性疾病中迈出了重要一步。Patisiran在其主要临床研究中表明,它可以阻止甚至改善大多数患者的这种疾病的潜在衰弱症状。基因沉默是一个很有前途的医学领域,看到这种科学转化为治疗方法帮助hATTR淀粉样变性患者是非常令人振奋的。"

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topicList=[TopicDto(id=8481, encryptionId=3da3848147, topicName=hATTR)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=20b5274, createdName=JR19860219, createdTime=Fri Jun 12 04:49:00 CST 2020, time=2020-06-12, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1972276, encodeId=cf1e19e227686, content=<a href='/topic/show?id=2b731e914ad' target=_blank style='color:#2F92EE;'>#TTR#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=25, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=17914, encryptionId=2b731e914ad, topicName=TTR)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c3ae503, createdName=pps20019, createdTime=Sat Mar 28 10:49:00 CST 2020, time=2020-03-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1289050, encodeId=b9b7128905051, content=<a href='/topic/show?id=077112e9237' target=_blank style='color:#2F92EE;'>#NICE#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=22, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=12792, encryptionId=077112e9237, topicName=NICE)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=743b215, createdName=fusion, createdTime=Fri Jul 12 00:49:00 CST 2019, time=2019-07-12, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1405172, encodeId=485114051e225, content=<a href='/topic/show?id=cb6e6591850' target=_blank style='color:#2F92EE;'>#淀粉样变#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=27, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=65918, encryptionId=cb6e6591850, topicName=淀粉样变)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=2ca02527521, createdName=xjy03, createdTime=Fri Jul 12 00:49:00 CST 2019, time=2019-07-12, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1434674, encodeId=a30014346e4f2, content=<a href='/topic/show?id=940265920ab' target=_blank style='color:#2F92EE;'>#淀粉样变性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=65920, encryptionId=940265920ab, topicName=淀粉样变性)], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/aLGWoFXAyMbIu3qymFOyheQLjPSX3OUs5GmkyBlcCOwTPIeq3why9NGibxxUqYo6hcx8qZLHZFgNPnBK1yzWeOFpyg2OnWOt0/0, createdBy=fa4716, createdName=zhouqu_8, createdTime=Fri Jul 12 00:49:00 CST 2019, time=2019-07-12, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1463684, encodeId=83a314636847c, content=<a href='/topic/show?id=7d27381288e' target=_blank style='color:#2F92EE;'>#变性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=24, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=38128, encryptionId=7d27381288e, topicName=变性)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f69f6545041, createdName=huangdf, createdTime=Fri Jul 12 00:49:00 CST 2019, time=2019-07-12, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1521633, encodeId=7a251521633d0, content=<a href='/topic/show?id=18bb8e08622' target=_blank style='color:#2F92EE;'>#英国NICE#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=36, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=87086, encryptionId=18bb8e08622, topicName=英国NICE)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=6ce711379167, createdName=lvygwyt2785, createdTime=Fri Jul 12 00:49:00 CST 2019, time=2019-07-12, status=1, ipAttribution=)]
    2020-06-12 JR19860219
  5. 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content=<a href='/topic/show?id=940265920ab' target=_blank style='color:#2F92EE;'>#淀粉样变性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=65920, encryptionId=940265920ab, topicName=淀粉样变性)], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/aLGWoFXAyMbIu3qymFOyheQLjPSX3OUs5GmkyBlcCOwTPIeq3why9NGibxxUqYo6hcx8qZLHZFgNPnBK1yzWeOFpyg2OnWOt0/0, createdBy=fa4716, createdName=zhouqu_8, createdTime=Fri Jul 12 00:49:00 CST 2019, time=2019-07-12, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1463684, encodeId=83a314636847c, content=<a href='/topic/show?id=7d27381288e' target=_blank style='color:#2F92EE;'>#变性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=24, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=38128, encryptionId=7d27381288e, topicName=变性)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f69f6545041, createdName=huangdf, createdTime=Fri Jul 12 00:49:00 CST 2019, time=2019-07-12, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1521633, encodeId=7a251521633d0, content=<a href='/topic/show?id=18bb8e08622' target=_blank style='color:#2F92EE;'>#英国NICE#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=36, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=87086, encryptionId=18bb8e08622, topicName=英国NICE)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=6ce711379167, createdName=lvygwyt2785, createdTime=Fri Jul 12 00:49:00 CST 2019, time=2019-07-12, status=1, ipAttribution=)]
    2020-03-28 pps20019
  6. [GetPortalCommentsPageByObjectIdResponse(id=2057663, encodeId=74e7205e66301, content=<a href='/topic/show?id=c02b106e29a' target=_blank style='color:#2F92EE;'>#LAM#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=10672, encryptionId=c02b106e29a, topicName=LAM)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=04ce158, createdName=lxg951, createdTime=Tue Mar 31 13:49:00 CST 2020, time=2020-03-31, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1704835, encodeId=51761e048355a, content=<a href='/topic/show?id=be56234245' target=_blank style='color:#2F92EE;'>#Alnylam#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=32, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=2342, encryptionId=be56234245, topicName=Alnylam)], attachment=null, authenticateStatus=null, createdAvatar=null, 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content=<a href='/topic/show?id=940265920ab' target=_blank style='color:#2F92EE;'>#淀粉样变性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=65920, encryptionId=940265920ab, topicName=淀粉样变性)], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/aLGWoFXAyMbIu3qymFOyheQLjPSX3OUs5GmkyBlcCOwTPIeq3why9NGibxxUqYo6hcx8qZLHZFgNPnBK1yzWeOFpyg2OnWOt0/0, createdBy=fa4716, createdName=zhouqu_8, createdTime=Fri Jul 12 00:49:00 CST 2019, time=2019-07-12, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1463684, encodeId=83a314636847c, content=<a href='/topic/show?id=7d27381288e' target=_blank style='color:#2F92EE;'>#变性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=24, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=38128, encryptionId=7d27381288e, topicName=变性)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f69f6545041, createdName=huangdf, createdTime=Fri Jul 12 00:49:00 CST 2019, time=2019-07-12, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1521633, encodeId=7a251521633d0, content=<a href='/topic/show?id=18bb8e08622' target=_blank style='color:#2F92EE;'>#英国NICE#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=36, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=87086, encryptionId=18bb8e08622, topicName=英国NICE)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=6ce711379167, createdName=lvygwyt2785, createdTime=Fri Jul 12 00:49:00 CST 2019, time=2019-07-12, status=1, ipAttribution=)]
    2019-07-12 fusion
  7. 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  8. 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  9. 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    2019-07-12 huangdf
  10. 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