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Ultragenyx宣布DTX401治疗Ia型糖原贮积病的阳性结果

2019-01-06 MedSci MedSci原创

Ia型糖原贮积病DTX401Ultragenyx
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Ultragenyx制药公司是一家生物制药公司，专注于开发用于严重罕见和超罕见遗传疾病的新产品，近日公布了正在进行的I/II期研究的低剂量组的正面安全性和有效性数据。

Ultragenyx制药公司是一家生物制药公司，专注于开发用于严重罕见和超罕见遗传疾病的新产品，近日公布了正在进行的I/II期研究的低剂量组的正面安全性和有效性数据。DTX401是一种基于腺相关病毒（AAV）的基因疗法，用于治疗Ia型糖原贮积病（GSDIa）。在所有三名患者中观察到生物反应，其通过改善的葡萄糖控制来反映，其中两名患者在受控的禁食挑战期间表现出临床上有意义的低血糖时间改善。

GSD属于一种合成、分解肝糖有缺陷的代谢疾病。造成肝糖储积症的病症有两种，即先天与后天。先天型肝糖储积症是因为出生前代谢系统出现问题（例如有缺陷的酵素）。整体而言，在不列颠哥伦比亚的调查显示，肝糖储积症发生率约为每10万名婴儿中会有2.3位，即每43000个婴儿中有1位；在美国的调查显示约20000至25000名婴儿中会有1人患有肝糖储积症；而在荷兰的发生率约为40000为婴儿中有1位。


原始出处：

http://www.firstwordpharma.com/node/1615895?tsid=4#axzz5bnF206kJ

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#插入话题
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    2019-02-21 bsmagic9140

    #TRA#

    0

  2. [GetPortalCommentsPageByObjectIdResponse(id=1657117, encodeId=4b56165e117bf, content=<a href='/topic/show?id=3ca31e71903' target=_blank style='color:#2F92EE;'>#TRA#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=23, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=17719, encryptionId=3ca31e71903, topicName=TRA)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=757b24868428, createdName=bsmagic9140, createdTime=Thu Feb 21 10:22:00 CST 2019, time=2019-02-21, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2042030, encodeId=f11b2042030ed, content=<a href='/topic/show?id=1a36181227e' target=_blank style='color:#2F92EE;'>#Ultragenyx#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=35, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=18122, encryptionId=1a36181227e, topicName=Ultragenyx)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=45b2276, createdName=839640778, createdTime=Sat Apr 13 05:22:00 CST 2019, time=2019-04-13, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1796564, encodeId=d4381e965642e, content=<a href='/topic/show?id=57211812121' target=_blank style='color:#2F92EE;'>#Ultra#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=27, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=18121, encryptionId=57211812121, topicName=Ultra)], attachment=null, authenticateStatus=null, createdAvatar=http://thirdqq.qlogo.cn/qqapp/101296147/385F510BDFE0F55A4FFDFDFFD9892943/100, createdBy=a3262500163, createdName=siiner, createdTime=Wed Aug 21 12:22:00 CST 2019, time=2019-08-21, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1642039, encodeId=483f164203968, content=<a href='/topic/show?id=679b9e514c8' target=_blank style='color:#2F92EE;'>#阳性结果#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=30, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=97514, encryptionId=679b9e514c8, topicName=阳性结果)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=28fb22837833, createdName=12498cb5m20暂无昵称, createdTime=Wed Mar 27 00:22:00 CST 2019, time=2019-03-27, status=1, ipAttribution=)]
    2019-04-13 839640778

    #Ultragenyx#

    0

  3. [GetPortalCommentsPageByObjectIdResponse(id=1657117, encodeId=4b56165e117bf, content=<a href='/topic/show?id=3ca31e71903' target=_blank style='color:#2F92EE;'>#TRA#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=23, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=17719, encryptionId=3ca31e71903, topicName=TRA)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=757b24868428, createdName=bsmagic9140, createdTime=Thu Feb 21 10:22:00 CST 2019, time=2019-02-21, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2042030, encodeId=f11b2042030ed, content=<a href='/topic/show?id=1a36181227e' target=_blank style='color:#2F92EE;'>#Ultragenyx#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=35, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=18122, encryptionId=1a36181227e, topicName=Ultragenyx)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=45b2276, createdName=839640778, createdTime=Sat Apr 13 05:22:00 CST 2019, time=2019-04-13, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1796564, encodeId=d4381e965642e, content=<a href='/topic/show?id=57211812121' target=_blank style='color:#2F92EE;'>#Ultra#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=27, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=18121, encryptionId=57211812121, topicName=Ultra)], attachment=null, authenticateStatus=null, createdAvatar=http://thirdqq.qlogo.cn/qqapp/101296147/385F510BDFE0F55A4FFDFDFFD9892943/100, createdBy=a3262500163, createdName=siiner, createdTime=Wed Aug 21 12:22:00 CST 2019, time=2019-08-21, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1642039, encodeId=483f164203968, content=<a href='/topic/show?id=679b9e514c8' target=_blank style='color:#2F92EE;'>#阳性结果#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=30, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=97514, encryptionId=679b9e514c8, topicName=阳性结果)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=28fb22837833, createdName=12498cb5m20暂无昵称, createdTime=Wed Mar 27 00:22:00 CST 2019, time=2019-03-27, status=1, ipAttribution=)]
    2019-08-21 siiner

    #Ultra#

    0

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    2019-03-27 12498cb5m20暂无昵称

    #阳性结果#

    0