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亨特氏综合症的治疗：DNL310取得孤儿药认定

2019-06-12 不详 网络

亨特氏综合症DNL310孤儿药
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Denali是一家开发神经退行性疾病疗法的生物制药公司，近日宣布，FDA授予了Denali的DNL310以孤儿药认定。

Denali是一家开发神经退行性疾病疗法的生物制药公司，近日宣布，FDA授予了Denali的DNL310以孤儿药认定。DNL310是一种重组形式的艾杜糖醛酸2-硫酸酯酶，DNL310使用了Denali专有的ETV技术，能够穿过血脑屏障，用以治疗粘多糖贮积症（亨特氏综合症）。

亨特氏综合症是由IDS酶的遗传缺陷引起的溶酶体贮积病。除了其他严重的临床表现外，大约三分之二的亨特综合征患者患有进行性认知障碍。DNL310在相关疾病模型中的临床前研究表明，与标准酶替代疗法相比，DNL310介导了中枢神经系统中的脑摄取增加并且下游疾病标志物出现显著减少。


原始出处：

http://www.firstwordpharma.com/node/1646675#axzz5qOw0cfEI

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  1. [GetPortalCommentsPageByObjectIdResponse(id=1634476, encodeId=51a116344e69d, content=<a href='/topic/show?id=03454543528' target=_blank style='color:#2F92EE;'>#孤儿药认定#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=29, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=45435, encryptionId=03454543528, topicName=孤儿药认定)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f66f22187170, createdName=dzx0922896, createdTime=Wed Jan 15 06:44:00 CST 2020, time=2020-01-15, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=367658, encodeId=22a136e658f8, content=向挑战病魔的科研人员致敬！向挑战病魔的科研人员致敬, beContent=null, objectType=article, channel=null, level=null, likeNumber=75, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=e5171668917, createdName=坚强007, createdTime=Wed Jun 12 21:04:57 CST 2019, time=2019-06-12, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=367641, encodeId=b67c36e64186, content=好, beContent=null, objectType=article, channel=null, level=null, likeNumber=60, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=afa32365525, createdName=122e052cm96暂无昵称, createdTime=Wed Jun 12 17:01:34 CST 2019, time=2019-06-12, status=1, ipAttribution=)]
    2020-01-15 dzx0922896

    #孤儿药认定#

    0

  2. [GetPortalCommentsPageByObjectIdResponse(id=1634476, encodeId=51a116344e69d, content=<a href='/topic/show?id=03454543528' target=_blank style='color:#2F92EE;'>#孤儿药认定#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=29, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=45435, encryptionId=03454543528, topicName=孤儿药认定)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f66f22187170, createdName=dzx0922896, createdTime=Wed Jan 15 06:44:00 CST 2020, time=2020-01-15, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=367658, encodeId=22a136e658f8, content=向挑战病魔的科研人员致敬！向挑战病魔的科研人员致敬, beContent=null, objectType=article, channel=null, level=null, likeNumber=75, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=e5171668917, createdName=坚强007, createdTime=Wed Jun 12 21:04:57 CST 2019, time=2019-06-12, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=367641, encodeId=b67c36e64186, content=好, beContent=null, objectType=article, channel=null, level=null, likeNumber=60, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=afa32365525, createdName=122e052cm96暂无昵称, createdTime=Wed Jun 12 17:01:34 CST 2019, time=2019-06-12, status=1, ipAttribution=)]
    2019-06-12 坚强007

    向挑战病魔的科研人员致敬！向挑战病魔的科研人员致敬

    0

  3. [GetPortalCommentsPageByObjectIdResponse(id=1634476, encodeId=51a116344e69d, content=<a href='/topic/show?id=03454543528' target=_blank style='color:#2F92EE;'>#孤儿药认定#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=29, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=45435, encryptionId=03454543528, topicName=孤儿药认定)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f66f22187170, createdName=dzx0922896, createdTime=Wed Jan 15 06:44:00 CST 2020, time=2020-01-15, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=367658, encodeId=22a136e658f8, content=向挑战病魔的科研人员致敬！向挑战病魔的科研人员致敬, beContent=null, objectType=article, channel=null, level=null, likeNumber=75, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=e5171668917, createdName=坚强007, createdTime=Wed Jun 12 21:04:57 CST 2019, time=2019-06-12, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=367641, encodeId=b67c36e64186, content=好, beContent=null, objectType=article, channel=null, level=null, likeNumber=60, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=afa32365525, createdName=122e052cm96暂无昵称, createdTime=Wed Jun 12 17:01:34 CST 2019, time=2019-06-12, status=1, ipAttribution=)]
    2019-06-12 122e052cm96暂无昵称

    好

    0