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基因疗法DTX301在鸟氨酸转氨甲酰酶缺乏症治疗中取得积极结果

2018-09-28 MedSci MedSci原创

DTX301鸟氨酸转氨甲酰酶缺乏症基因疗法
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Ultragenyx制药公司是一家专注于开发超罕见疾病药物的生物制药公司。Ultragenyx近日公布了有关基因疗法DTX301的积极的安全性和有效性数据，DTX301是一种用于治疗鸟氨酸转氨甲酰酶（OTC）缺陷的腺相关病毒（AAV）基因疗法。

Ultragenyx制药公司是一家专注于开发超罕见疾病药物的生物制药公司。Ultragenyx近日公布了有关基因疗法DTX301的积极的安全性和有效性数据，DTX301是一种用于治疗鸟氨酸转氨甲酰酶（OTC）缺陷的腺相关病毒（AAV）基因疗法。

鸟胺酸氨甲酰基转移酶缺乏症（OTC）为先天性遗传代谢性罕见病，患者存在尿素循环酶缺陷，患者尿素循环中断，血氨增高，血瓜氨酸、精氨酸降低，谷氨酸酰胺、丙氨酸升高，而尿乳清酸和嘧啶排泄增加。重症患儿常在出生后数日内发生呕吐、拒食、嗜睡、惊厥、肌张力低下、昏迷等症状；轻症患儿则表现为间隙性反复发作，嗜睡、易激惹、不宁、共济失调等。

Ultragenyx首席医疗官Eric Crombez博士说：“来自前两个剂量组的数据继续表现出可接受的安全性和临床活动证据，其中1名患者在24周时实现了尿素生成的正常化”。


原始出处：

http://www.firstwordpharma.com/node/1593717#axzz5SJBcSQbc

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    2018-12-02 jyzxjiangqin

    基因疗法中的影响。

    0

  2. [GetPortalCommentsPageByObjectIdResponse(id=354277, encodeId=c0d43542e7de, content=基因疗法中的影响。, beContent=null, objectType=article, channel=null, level=null, likeNumber=64, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=http://cacheapi.medsci.cn/resource/upload/20160806/IMG57A5698BDB0244113.jpg, createdBy=32a91358059, createdName=jyzxjiangqin, createdTime=Sun Dec 02 17:45:53 CST 2018, time=2018-12-02, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=349854, encodeId=32b034985461, content=基因疗法取得积极结果。, beContent=null, objectType=article, channel=null, level=null, likeNumber=61, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=http://cacheapi.medsci.cn/resource/upload/20160806/IMG57A5698BDB0244113.jpg, createdBy=32a91358059, createdName=jyzxjiangqin, createdTime=Fri Oct 19 17:45:10 CST 2018, time=2018-10-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=347187, encodeId=923634e18739, content=了解一下谢谢, beContent=null, objectType=article, channel=null, level=null, likeNumber=63, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=http://thirdwx.qlogo.cn/mmopen/vi_32/Q0j4TwGTfTLbTp9nZtjwVrSZNxAXbAicnKQWnic2eBdJUQYhxO36XdkstSicr7FVF8p5ReH4omtmsbnLdvZLiaJJ1A/132, createdBy=8bff2327056, createdName=kafei, createdTime=Sat Sep 29 08:14:52 CST 2018, time=2018-09-29, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=347162, encodeId=1aa734e1626c, content=不错的文章值得推荐一下, beContent=null, objectType=article, channel=null, level=null, likeNumber=59, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=04f22055142, createdName=131****2916, createdTime=Fri Sep 28 23:13:06 CST 2018, time=2018-09-28, status=1, ipAttribution=)]
    2018-10-19 jyzxjiangqin

    基因疗法取得积极结果。

    0

  3. [GetPortalCommentsPageByObjectIdResponse(id=354277, encodeId=c0d43542e7de, content=基因疗法中的影响。, beContent=null, objectType=article, channel=null, level=null, likeNumber=64, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=http://cacheapi.medsci.cn/resource/upload/20160806/IMG57A5698BDB0244113.jpg, createdBy=32a91358059, createdName=jyzxjiangqin, createdTime=Sun Dec 02 17:45:53 CST 2018, time=2018-12-02, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=349854, encodeId=32b034985461, content=基因疗法取得积极结果。, beContent=null, objectType=article, channel=null, level=null, likeNumber=61, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=http://cacheapi.medsci.cn/resource/upload/20160806/IMG57A5698BDB0244113.jpg, createdBy=32a91358059, createdName=jyzxjiangqin, createdTime=Fri Oct 19 17:45:10 CST 2018, time=2018-10-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=347187, encodeId=923634e18739, content=了解一下谢谢, beContent=null, objectType=article, channel=null, level=null, likeNumber=63, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=http://thirdwx.qlogo.cn/mmopen/vi_32/Q0j4TwGTfTLbTp9nZtjwVrSZNxAXbAicnKQWnic2eBdJUQYhxO36XdkstSicr7FVF8p5ReH4omtmsbnLdvZLiaJJ1A/132, createdBy=8bff2327056, createdName=kafei, createdTime=Sat Sep 29 08:14:52 CST 2018, time=2018-09-29, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=347162, encodeId=1aa734e1626c, content=不错的文章值得推荐一下, beContent=null, objectType=article, channel=null, level=null, likeNumber=59, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=04f22055142, createdName=131****2916, createdTime=Fri Sep 28 23:13:06 CST 2018, time=2018-09-28, status=1, ipAttribution=)]
    2018-09-29 kafei

    了解一下谢谢

    0

  4. [GetPortalCommentsPageByObjectIdResponse(id=354277, encodeId=c0d43542e7de, content=基因疗法中的影响。, beContent=null, objectType=article, channel=null, level=null, likeNumber=64, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=http://cacheapi.medsci.cn/resource/upload/20160806/IMG57A5698BDB0244113.jpg, createdBy=32a91358059, createdName=jyzxjiangqin, createdTime=Sun Dec 02 17:45:53 CST 2018, time=2018-12-02, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=349854, encodeId=32b034985461, content=基因疗法取得积极结果。, beContent=null, objectType=article, channel=null, level=null, likeNumber=61, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=http://cacheapi.medsci.cn/resource/upload/20160806/IMG57A5698BDB0244113.jpg, createdBy=32a91358059, createdName=jyzxjiangqin, createdTime=Fri Oct 19 17:45:10 CST 2018, time=2018-10-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=347187, encodeId=923634e18739, content=了解一下谢谢, beContent=null, objectType=article, channel=null, level=null, likeNumber=63, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=http://thirdwx.qlogo.cn/mmopen/vi_32/Q0j4TwGTfTLbTp9nZtjwVrSZNxAXbAicnKQWnic2eBdJUQYhxO36XdkstSicr7FVF8p5ReH4omtmsbnLdvZLiaJJ1A/132, createdBy=8bff2327056, createdName=kafei, createdTime=Sat Sep 29 08:14:52 CST 2018, time=2018-09-29, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=347162, encodeId=1aa734e1626c, content=不错的文章值得推荐一下, beContent=null, objectType=article, channel=null, level=null, likeNumber=59, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=04f22055142, createdName=131****2916, createdTime=Fri Sep 28 23:13:06 CST 2018, time=2018-09-28, status=1, ipAttribution=)]
    2018-09-28 131****2916

    不错的文章值得推荐一下

    0