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2019年遗传代谢紊乱学会年会：Pegzilarginase治疗精氨酸酶基因缺失患者的阳性I/II期临床数据

2019-04-08 不详 网络

精氨酸酶基因遗传代谢紊乱Pegzilarginase
关注

Aeglea是一家临床阶段的生物技术公司，致力于为罕见的遗传性疾病和癌症患者设计和开发创新的酶疗法，近日宣布将在2019年遗传性代谢紊乱学会（SIMD）年会上公布pegzilarginase治疗精氨酸酶基因缺失的最新I/II期临床的阳性数据。

Aeglea是一家临床阶段的生物技术公司，致力于为罕见的遗传性疾病和癌症患者设计和开发创新的酶疗法，近日宣布将在2019年遗传性代谢紊乱学会（SIMD）年会上公布pegzilarginase治疗精氨酸酶基因缺失的最新I/II期临床的阳性数据。精氨酸酶是催化水解L-精氨酸生成鸟氨酸与尿素的反应酶，如果精氨酸酶基因缺失，患者血浆中精氨酸浓度将异常升高。

I/II期临床证明了pegzilarginase可持续降低血浆精氨酸浓度，且患者对治疗通常耐受良好。超敏反应很少发生，即使发生也可通过标准手段进行控制，因此不会导致治疗中止。


原始出处：

http://www.firstwordpharma.com/node/1633819?tsid=4#axzz5kSaSXmAK

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    2020-01-02 chenlianhui

    #临床数据#

    0

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    2020-02-06 gostraight

    #精氨酸#

    0

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    2020-02-20 heli0118

    #基因缺失#

    0

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    2019-10-24 feather89

    #PE#

    0

  5. [GetPortalCommentsPageByObjectIdResponse(id=2016725, encodeId=2da82016e2504, content=<a href='/topic/show?id=75a2228481a' target=_blank style='color:#2F92EE;'>#临床数据#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=23, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=22848, encryptionId=75a2228481a, topicName=临床数据)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=fa4f352, createdName=chenlianhui, createdTime=Thu Jan 02 17:59:00 CST 2020, time=2020-01-02, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1928760, encodeId=c1701928e603d, content=<a href='/topic/show?id=3ee5e6449ff' target=_blank style='color:#2F92EE;'>#精氨酸#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=36, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=76449, encryptionId=3ee5e6449ff, topicName=精氨酸)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=db5b227, createdName=gostraight, createdTime=Thu Feb 06 02:59:00 CST 2020, time=2020-02-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1987041, encodeId=9261198e041e0, content=<a href='/topic/show?id=8ea3418e3d4' target=_blank style='color:#2F92EE;'>#基因缺失#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=36, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=41873, encryptionId=8ea3418e3d4, topicName=基因缺失)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=21015, createdName=heli0118, createdTime=Thu Feb 20 23:59:00 CST 2020, time=2020-02-20, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1727961, encodeId=38ed1e27961d1, content=<a href='/topic/show?id=986913980dd' target=_blank style='color:#2F92EE;'>#PE#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=27, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=13980, encryptionId=986913980dd, topicName=PE)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f06c33759375, createdName=feather89, createdTime=Thu Oct 24 05:59:00 CST 2019, time=2019-10-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1652883, encodeId=503d1652883cf, content=<a href='/topic/show?id=5b011018e07' target=_blank style='color:#2F92EE;'>#I期临床#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=21, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=10187, encryptionId=5b011018e07, topicName=I期临床)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=fc1f24335670, createdName=医生2402, createdTime=Thu Apr 11 22:59:00 CST 2019, time=2019-04-11, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1515331, encodeId=a5a91515331ba, content=<a href='/topic/show?id=a9d2802479' target=_blank style='color:#2F92EE;'>#GINA#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=28, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=8024, encryptionId=a9d2802479, topicName=GINA)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=cd0110767805, createdName=ms5880526374914242, createdTime=Tue Apr 09 23:59:00 CST 2019, time=2019-04-09, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1524692, encodeId=68ca15246929b, content=<a href='/topic/show?id=4ba395994d' target=_blank style='color:#2F92EE;'>#II期临床#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=18, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9599, encryptionId=4ba395994d, topicName=II期临床)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=139811614978, createdName=guihongzh, createdTime=Tue Apr 09 23:59:00 CST 2019, time=2019-04-09, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1533495, encodeId=1256153349531, content=<a href='/topic/show?id=e59c1400060' target=_blank style='color:#2F92EE;'>#PEG#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=0, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=14000, encryptionId=e59c1400060, topicName=PEG)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=bbc812457783, createdName=wleon8899, createdTime=Tue Apr 09 23:59:00 CST 2019, time=2019-04-09, status=1, ipAttribution=)]
    2019-04-11 医生2402

    #I期临床#

    0

  6. [GetPortalCommentsPageByObjectIdResponse(id=2016725, encodeId=2da82016e2504, content=<a href='/topic/show?id=75a2228481a' target=_blank style='color:#2F92EE;'>#临床数据#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=23, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=22848, encryptionId=75a2228481a, topicName=临床数据)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=fa4f352, createdName=chenlianhui, createdTime=Thu Jan 02 17:59:00 CST 2020, time=2020-01-02, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1928760, encodeId=c1701928e603d, content=<a href='/topic/show?id=3ee5e6449ff' target=_blank style='color:#2F92EE;'>#精氨酸#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=36, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=76449, encryptionId=3ee5e6449ff, topicName=精氨酸)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=db5b227, createdName=gostraight, createdTime=Thu Feb 06 02:59:00 CST 2020, time=2020-02-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1987041, encodeId=9261198e041e0, content=<a href='/topic/show?id=8ea3418e3d4' target=_blank style='color:#2F92EE;'>#基因缺失#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=36, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=41873, encryptionId=8ea3418e3d4, topicName=基因缺失)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=21015, createdName=heli0118, createdTime=Thu Feb 20 23:59:00 CST 2020, time=2020-02-20, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1727961, encodeId=38ed1e27961d1, content=<a href='/topic/show?id=986913980dd' target=_blank style='color:#2F92EE;'>#PE#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=27, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=13980, encryptionId=986913980dd, topicName=PE)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f06c33759375, createdName=feather89, createdTime=Thu Oct 24 05:59:00 CST 2019, time=2019-10-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1652883, encodeId=503d1652883cf, content=<a href='/topic/show?id=5b011018e07' target=_blank style='color:#2F92EE;'>#I期临床#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=21, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=10187, encryptionId=5b011018e07, topicName=I期临床)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=fc1f24335670, createdName=医生2402, createdTime=Thu Apr 11 22:59:00 CST 2019, time=2019-04-11, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1515331, encodeId=a5a91515331ba, content=<a href='/topic/show?id=a9d2802479' target=_blank style='color:#2F92EE;'>#GINA#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=28, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=8024, encryptionId=a9d2802479, topicName=GINA)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=cd0110767805, createdName=ms5880526374914242, createdTime=Tue Apr 09 23:59:00 CST 2019, time=2019-04-09, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1524692, encodeId=68ca15246929b, content=<a href='/topic/show?id=4ba395994d' target=_blank style='color:#2F92EE;'>#II期临床#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=18, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9599, encryptionId=4ba395994d, topicName=II期临床)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=139811614978, createdName=guihongzh, createdTime=Tue Apr 09 23:59:00 CST 2019, time=2019-04-09, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1533495, encodeId=1256153349531, content=<a href='/topic/show?id=e59c1400060' target=_blank style='color:#2F92EE;'>#PEG#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=0, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=14000, encryptionId=e59c1400060, topicName=PEG)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=bbc812457783, createdName=wleon8899, createdTime=Tue Apr 09 23:59:00 CST 2019, time=2019-04-09, status=1, ipAttribution=)]
    2019-04-09 ms5880526374914242

    #GINA#

    0

  7. [GetPortalCommentsPageByObjectIdResponse(id=2016725, encodeId=2da82016e2504, content=<a href='/topic/show?id=75a2228481a' target=_blank style='color:#2F92EE;'>#临床数据#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=23, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=22848, encryptionId=75a2228481a, topicName=临床数据)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=fa4f352, createdName=chenlianhui, createdTime=Thu Jan 02 17:59:00 CST 2020, time=2020-01-02, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1928760, encodeId=c1701928e603d, content=<a href='/topic/show?id=3ee5e6449ff' target=_blank style='color:#2F92EE;'>#精氨酸#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=36, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=76449, encryptionId=3ee5e6449ff, topicName=精氨酸)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=db5b227, createdName=gostraight, createdTime=Thu Feb 06 02:59:00 CST 2020, time=2020-02-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1987041, encodeId=9261198e041e0, content=<a href='/topic/show?id=8ea3418e3d4' target=_blank style='color:#2F92EE;'>#基因缺失#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=36, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=41873, encryptionId=8ea3418e3d4, topicName=基因缺失)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=21015, createdName=heli0118, createdTime=Thu Feb 20 23:59:00 CST 2020, time=2020-02-20, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1727961, encodeId=38ed1e27961d1, content=<a href='/topic/show?id=986913980dd' target=_blank style='color:#2F92EE;'>#PE#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=27, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=13980, encryptionId=986913980dd, topicName=PE)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f06c33759375, createdName=feather89, createdTime=Thu Oct 24 05:59:00 CST 2019, time=2019-10-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1652883, encodeId=503d1652883cf, content=<a href='/topic/show?id=5b011018e07' target=_blank style='color:#2F92EE;'>#I期临床#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=21, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=10187, encryptionId=5b011018e07, topicName=I期临床)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=fc1f24335670, createdName=医生2402, createdTime=Thu Apr 11 22:59:00 CST 2019, time=2019-04-11, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1515331, encodeId=a5a91515331ba, content=<a href='/topic/show?id=a9d2802479' target=_blank style='color:#2F92EE;'>#GINA#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=28, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=8024, encryptionId=a9d2802479, topicName=GINA)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=cd0110767805, createdName=ms5880526374914242, createdTime=Tue Apr 09 23:59:00 CST 2019, time=2019-04-09, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1524692, encodeId=68ca15246929b, content=<a href='/topic/show?id=4ba395994d' target=_blank style='color:#2F92EE;'>#II期临床#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=18, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9599, encryptionId=4ba395994d, topicName=II期临床)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=139811614978, createdName=guihongzh, createdTime=Tue Apr 09 23:59:00 CST 2019, time=2019-04-09, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1533495, encodeId=1256153349531, content=<a href='/topic/show?id=e59c1400060' target=_blank style='color:#2F92EE;'>#PEG#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=0, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=14000, encryptionId=e59c1400060, topicName=PEG)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=bbc812457783, createdName=wleon8899, createdTime=Tue Apr 09 23:59:00 CST 2019, time=2019-04-09, status=1, ipAttribution=)]
    2019-04-09 guihongzh

    #II期临床#

    0

  8. [GetPortalCommentsPageByObjectIdResponse(id=2016725, encodeId=2da82016e2504, content=<a href='/topic/show?id=75a2228481a' target=_blank style='color:#2F92EE;'>#临床数据#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=23, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=22848, encryptionId=75a2228481a, topicName=临床数据)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=fa4f352, createdName=chenlianhui, createdTime=Thu Jan 02 17:59:00 CST 2020, time=2020-01-02, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1928760, encodeId=c1701928e603d, content=<a href='/topic/show?id=3ee5e6449ff' target=_blank style='color:#2F92EE;'>#精氨酸#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=36, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=76449, encryptionId=3ee5e6449ff, topicName=精氨酸)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=db5b227, createdName=gostraight, createdTime=Thu Feb 06 02:59:00 CST 2020, time=2020-02-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1987041, encodeId=9261198e041e0, content=<a href='/topic/show?id=8ea3418e3d4' target=_blank style='color:#2F92EE;'>#基因缺失#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=36, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=41873, encryptionId=8ea3418e3d4, topicName=基因缺失)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=21015, createdName=heli0118, createdTime=Thu Feb 20 23:59:00 CST 2020, time=2020-02-20, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1727961, encodeId=38ed1e27961d1, content=<a href='/topic/show?id=986913980dd' target=_blank style='color:#2F92EE;'>#PE#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=27, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=13980, encryptionId=986913980dd, topicName=PE)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f06c33759375, createdName=feather89, createdTime=Thu Oct 24 05:59:00 CST 2019, time=2019-10-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1652883, encodeId=503d1652883cf, content=<a href='/topic/show?id=5b011018e07' target=_blank style='color:#2F92EE;'>#I期临床#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=21, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=10187, encryptionId=5b011018e07, topicName=I期临床)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=fc1f24335670, createdName=医生2402, createdTime=Thu Apr 11 22:59:00 CST 2019, time=2019-04-11, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1515331, encodeId=a5a91515331ba, content=<a href='/topic/show?id=a9d2802479' target=_blank style='color:#2F92EE;'>#GINA#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=28, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=8024, encryptionId=a9d2802479, topicName=GINA)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=cd0110767805, createdName=ms5880526374914242, createdTime=Tue Apr 09 23:59:00 CST 2019, time=2019-04-09, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1524692, encodeId=68ca15246929b, content=<a href='/topic/show?id=4ba395994d' target=_blank style='color:#2F92EE;'>#II期临床#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=18, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9599, encryptionId=4ba395994d, topicName=II期临床)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=139811614978, createdName=guihongzh, createdTime=Tue Apr 09 23:59:00 CST 2019, time=2019-04-09, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1533495, encodeId=1256153349531, content=<a href='/topic/show?id=e59c1400060' target=_blank style='color:#2F92EE;'>#PEG#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=0, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=14000, encryptionId=e59c1400060, topicName=PEG)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=bbc812457783, createdName=wleon8899, createdTime=Tue Apr 09 23:59:00 CST 2019, time=2019-04-09, status=1, ipAttribution=)]
    2019-04-09 wleon8899

    #PEG#

    0