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FDA通过了一项遗传性佝偻病的治疗方法

2018-04-19 MedSci MedSci原创

遗传性佝偻病
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美国的监管机构已经批准了第一种治疗成人和儿童与x相关联的低磷血症(XLH)的药物，这是一种罕见的遗传性佝偻病。XLH导致血液中磷含量低，导致儿童和青少年骨骼生长和发育受损，以及患者一生中骨矿化问题。Ultragenyx制药公司和Kyowa Hakko Kirin的Crysvita (burosumab-twza)是一种能阻止成纤维细胞生长因子23 (FGF23)的抗体，该激素能引起磷酸盐尿排泄，并

美国的监管机构已经批准了第一种治疗成人和儿童与x相关联的低磷血症(XLH)的药物，这是一种罕见的遗传性佝偻病。XLH导致血液中磷含量低，导致儿童和青少年骨骼生长和发育受损，以及患者一生中骨矿化问题。Ultragenyx制药公司和Kyowa Hakko Kirin的Crysvita (burosumab-twza)是一种能阻止成纤维细胞生长因子23 (FGF23)的抗体，该激素能引起磷酸盐尿排泄，并抑制肾脏的活性维生素D的产生。"批准Crysvita x连锁低磷酸盐血患者来说确实是一个转折点，因为它是第一个针对纠正肾磷酸盐消耗的治疗方法，" Tom Carpenter说到，他是该项研究负责人， x连锁低磷酸盐血耶鲁中心主任，耶鲁大学医学院的儿科内分泌学教授。"通过靶向这一机制，Crysvita会导致磷酸盐代谢的持续改善，并同时修复骨骼，即使是在常规治疗之前。""最重要的是，Crysvita的给药方案比现有的治疗方法要轻得多，并且应该很容易被家庭接受。我期望它能彻底改变XLH患者的治疗方法。"原文出处：http：//www.pharmatimes.com/news/fda_clears_f

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    2018-06-03 般若傻瓜

    #遗传性#

    0

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    2018-04-23 惠映实验室

    学习了.谢谢分享.

    0

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    2018-04-23 yfjms

    学习

    0

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    2018-04-21 luckyshitiancai_42705684

    #佝偻病#

    0

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    2018-04-21 showtest

    #治疗方法#

    0

  6. [GetPortalCommentsPageByObjectIdResponse(id=1860037, encodeId=1d3e186003e61, content=<a href='/topic/show?id=cc73951054b' target=_blank style='color:#2F92EE;'>#遗传性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=17, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=95105, encryptionId=cc73951054b, topicName=遗传性)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=ad9f79, createdName=般若傻瓜, createdTime=Sun Jun 03 21:14:00 CST 2018, time=2018-06-03, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=308535, encodeId=36dd3085359f, content=学习了.谢谢分享., beContent=null, objectType=article, channel=null, level=null, likeNumber=57, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/xkvRjYnJzzRJ2R0y7VhicibvkjPWHjIMmu4ZpKnE9cWAu4lVicS3uDUCfF4M4JWvrkrYfpb2KehPV0Ee29tPqyPEebBUibiaKhnvD/0, createdBy=f8412001184, createdName=惠映实验室, createdTime=Mon Apr 23 12:28:40 CST 2018, time=2018-04-23, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=308410, encodeId=3ce3308410db, content=学习, beContent=null, objectType=article, channel=null, level=null, likeNumber=65, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c3da1990174, createdName=yfjms, createdTime=Mon Apr 23 06:53:37 CST 2018, time=2018-04-23, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1341361, encodeId=29f0134136133, content=<a href='/topic/show?id=001d26e84b9' target=_blank style='color:#2F92EE;'>#佝偻病#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=30, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=26784, encryptionId=001d26e84b9, topicName=佝偻病)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=6eb32500176, createdName=luckyshitiancai_42705684, createdTime=Sat Apr 21 12:14:00 CST 2018, time=2018-04-21, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1399246, encodeId=1a5d139924661, content=<a href='/topic/show?id=cac364650c0' target=_blank style='color:#2F92EE;'>#治疗方法#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=18, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=64650, encryptionId=cac364650c0, topicName=治疗方法)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=8cec2200006, createdName=showtest, createdTime=Sat Apr 21 12:14:00 CST 2018, time=2018-04-21, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=307732, encodeId=294930e73232, content=学习学习学习, beContent=null, objectType=article, channel=null, level=null, likeNumber=53, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://cdnapi.center.medsci.cn/medsci/head/2018/03/27/6f2d63c799d68129300901eb0e962dd4.jpg, createdBy=ddf12178538, createdName=小贝熊, createdTime=Sat Apr 21 00:19:47 CST 2018, time=2018-04-21, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=307368, encodeId=7eec30e3686a, content=学习一下谢谢分享, beContent=null, objectType=article, channel=null, level=null, likeNumber=59, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/jW482SpianMayicTRbRZ5RzaiabIFicf63hKvVMjL6aJlx4yX4UcXmzX9DyibCRcicq0QtCRWMaQKh8MYjE1D3EKUBopgiaGWYuqbeR/0, createdBy=0c2b1999806, createdName=三生有幸9135, createdTime=Thu Apr 19 22:12:30 CST 2018, time=2018-04-19, status=1, ipAttribution=)]
    2018-04-21 小贝熊

    学习学习学习

    0

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    2018-04-19 三生有幸9135

    学习一下谢谢分享

    0