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ENDO 2016：Strensiq可改善低磷酸酯酶症患者的骨愈合情况

2016-04-18 MedSci MedSci原创

低磷酸血症骨愈合酶替代疗法
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据内分泌学会2016年度会议上展示的一项新的研究表明，使用strensiq治疗围产期及婴儿期低磷酸酯酶症可改善患儿的骨愈合和身体功能，且效果可持续3年以上。

据内分泌学会2016年度会议上展示的一项新的研究表明，使用strensiq治疗围产期及婴儿期低磷酸酯酶症可改善患儿的骨愈合和身体功能，且效果可持续3年以上。

低磷酸酯酶症（HPP）是一种罕见的代谢性疾病，其特点是碱性磷酸酶活性较低和骨矿化缺陷导致骨骼畸形和其他的骨骼发育异常，以及肌肉无力、抽搐、疼痛和呼吸衰竭。

此研究发现来自于一项正在进行的非盲Strensiq 2期临床试验的研究结果。该研究中，研究人员发现经Strensiq 治疗5年，11-17岁的青少年HPP患者（n = 12）在骨愈合（放射学评定）和生理功能均得到了改善。此外，新的临时结果显示HHP患者体内积累的非特异性碱性磷酸酶（TNSALP）在两个关键组织中减少，而且青少年和成年HPP患者的生理功能均得到了改善（基线时期13-66岁）。

“ENDO上所展示的该项研究结果表明， 经Strensiq 治疗后HPP儿童患者的各项关键评估标准均得到了具有临床意义的明显改善，且效果持久，这些评估措施包括骨愈合，肢体功能和疼痛，” Alexion公司全球研发主管和执行副总裁，Martin Mackay博士在新闻报道中说道，“该研究数据为strensiq治疗的长期疗效和安全性提供了额外的信息，Strensiq是一种酶替代疗法，其治疗是针对引起低磷酸酯酶症的病因，即通过替换缺陷的TNSALP酶来达到治疗的目的。”

FDA已批准Strensiq用于治疗治疗围产期、婴儿及青少年HP。

原始出处：

Liese J, et al. Strensiq improves bone healing in children with hypophosphatasia. Healio, April 17, 2016.

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    2016-09-01 ylzr123

    好文，值得学习，赞！

    0

  2. [GetPortalCommentsPageByObjectIdResponse(id=108728, encodeId=200c108e282d, content=好文，值得学习，赞！, beContent=null, objectType=article, channel=null, level=null, likeNumber=65, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/NUyjXTCJjo7s7ZCKOmmbBTD6nE7gcd5OZpvyGEzkxBP3S275k5ZJVrsPkotZhBglRHM9rKvcj1OgkssqhcnoPw8zIaMZTF8c/0, createdBy=4c441942088, createdName=ylzr123, createdTime=Thu Sep 01 08:52:00 CST 2016, time=2016-09-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2026974, encodeId=cb9120269e405, content=<a href='/topic/show?id=921d101668c8' target=_blank style='color:#2F92EE;'>#骨愈合#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=29, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=101668, encryptionId=921d101668c8, topicName=骨愈合)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=85aa264, createdName=redcrab, createdTime=Sun Aug 07 21:00:00 CST 2016, time=2016-08-07, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=79408, encodeId=c268e940840, content=文章不错, beContent=null, objectType=article, channel=null, level=null, likeNumber=98, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=240a1683959, createdName=李继凯, createdTime=Tue Apr 19 00:39:00 CST 2016, time=2016-04-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=79409, encodeId=c8cce940976, content=值得关注, beContent=null, objectType=article, channel=null, level=null, likeNumber=100, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=240a1683959, createdName=李继凯, createdTime=Tue Apr 19 00:39:00 CST 2016, time=2016-04-19, status=1, ipAttribution=)]
    2016-08-07 redcrab

    #骨愈合#

    0

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    2016-04-19 李继凯

    文章不错

    0

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    2016-04-19 李继凯

    值得关注

    0