RNAi疗法lumasiran:获得英国MHRA支持治疗1型原发性高草酸尿症

2020-07-16 MedSci原创 MedSci原创

Lumasiran是通过皮下给药、靶向羟基酸氧化酶1(HAO1)的RNAi治疗药物。

英国药品和保健产品监管局(MHRA)支持将Alnylam的lumasiran,用于治疗患有极罕见疾病的1型原发性高草酸尿症(PH1)患者。

PH1作为一种罕见疾病,在英国大约有90位患者,疾病特征为肝脏毒性草酸盐的异常堆积,从而引起疼痛和复发性肾结石,并最终对肾脏和其他重要器官造成不可挽回的伤害。

目前没有针对该病的治疗选择,现有的策略集中在疾病管理上,晚期疾病患者需要进行肾脏透析,直到他们有能力并有资格接受双肾/肝移植。

See the source image

图片来源:https://seekingalpha.com/article/4199538-alnylam-pharmaceuticals-alny-lumasarin-trial-data-slideshow

Lumasiran是一种皮下给药、靶向羟基酸氧化酶1(HAO1)的RNAi治疗药物。通过沉默HAO1并消耗乙醇酸氧化酶(GO),Lumasiran可抑制草酸盐的产生,草酸盐是直接影响PH1病理生理的代谢产物。

来自ILLUMINATE-A试验的临床数据表明,该药物相对于基线水平可使草酸尿平均减少65.4%,与安慰剂相比减少了53.5%。

Alnylam英国和爱尔兰大区经理Brendan Martin表示:“MHRA对lumasiran的积极意见,对于目前治疗选择有限的PH1患者及其家人来说,是个好消息。该类患者迫切需要能够解决根本问题的新药。这一决定将使英国的PH1患者尽早获得lumasiran。”

目前该药物正在欧洲和美国的监管机构进行审查。

原始出处:

http://www.pharmatimes.com/news/uks_mhra_allows_early_access_to_lumasiran_for_ultra-rare_disease_1344982

版权声明:
本网站所有内容来源注明为“梅斯医学”或“MedSci原创”的文字、图片和音视频资料,版权均属于梅斯医学所有。非经授权,任何媒体、网站或个人不得转载,授权转载时须注明来源为“梅斯医学”。其它来源的文章系转载文章,或“梅斯号”自媒体发布的文章,仅系出于传递更多信息之目的,本站仅负责审核内容合规,其内容不代表本站立场,本站不负责内容的准确性和版权。如果存在侵权、或不希望被转载的媒体或个人可与我们联系,我们将立即进行删除处理。
在此留言
评论区 (7)
#插入话题
  1. [GetPortalCommentsPageByObjectIdResponse(id=1846915, encodeId=330e1846915ad, content=<a href='/topic/show?id=d3a33e0306d' target=_blank style='color:#2F92EE;'>#原发性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=26, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=37030, encryptionId=d3a33e0306d, topicName=原发性)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=8e4c53, createdName=jiyangfei, createdTime=Fri Jan 29 20:20:29 CST 2021, time=2021-01-29, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1648488, encodeId=4d9316484884b, content=<a href='/topic/show?id=c3b68e084ff' target=_blank style='color:#2F92EE;'>#英国MHRA#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=28, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=87084, encryptionId=c3b68e084ff, topicName=英国MHRA)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=e32f23584568, createdName=minzju5060, createdTime=Fri Mar 19 02:20:29 CST 2021, time=2021-03-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1751683, encodeId=a7511e51683dd, content=<a href='/topic/show?id=2c2c1553e56' target=_blank style='color:#2F92EE;'>#RNAi#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=15537, encryptionId=2c2c1553e56, topicName=RNAi)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=d9d436366397, createdName=yinxm8315, createdTime=Sun Apr 04 12:20:29 CST 2021, time=2021-04-04, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1921064, encodeId=4a46192106444, content=<a href='/topic/show?id=921b5e08167' target=_blank style='color:#2F92EE;'>#支持治疗#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=29, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=57081, encryptionId=921b5e08167, topicName=支持治疗)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=640c454, createdName=qblt, createdTime=Sat Aug 29 00:20:29 CST 2020, time=2020-08-29, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=803539, encodeId=21d5803539e9, content=原发性高草酸, beContent=null, objectType=article, channel=null, level=null, likeNumber=87, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20200720/956eb01f4b594ee783debae7a44d9096/e569cb8381764328b493c23491d86c27.jpg, createdBy=2cbc5401622, createdName=老婆子, createdTime=Mon Jul 20 20:20:22 CST 2020, time=2020-07-20, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=803216, encodeId=dfad803216c9, content=厉害, beContent=null, objectType=article, channel=null, level=null, likeNumber=89, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=27d45175397, createdName=146e617dm26暂无昵称, createdTime=Sat Jul 18 21:27:37 CST 2020, time=2020-07-18, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1536870, encodeId=7d1215368e016, content=<a href='/topic/show?id=7cad1554076' target=_blank style='color:#2F92EE;'>#RNAi疗法Lumasiran#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=28, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=15540, encryptionId=7cad1554076, topicName=RNAi疗法Lumasiran)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=fe1712732896, createdName=fzwish20004, createdTime=Sat Jul 18 10:20:29 CST 2020, time=2020-07-18, status=1, ipAttribution=)]
  2. [GetPortalCommentsPageByObjectIdResponse(id=1846915, encodeId=330e1846915ad, content=<a href='/topic/show?id=d3a33e0306d' target=_blank style='color:#2F92EE;'>#原发性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=26, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=37030, encryptionId=d3a33e0306d, topicName=原发性)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=8e4c53, createdName=jiyangfei, createdTime=Fri Jan 29 20:20:29 CST 2021, time=2021-01-29, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1648488, encodeId=4d9316484884b, content=<a href='/topic/show?id=c3b68e084ff' target=_blank style='color:#2F92EE;'>#英国MHRA#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=28, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=87084, encryptionId=c3b68e084ff, topicName=英国MHRA)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=e32f23584568, createdName=minzju5060, createdTime=Fri Mar 19 02:20:29 CST 2021, time=2021-03-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1751683, encodeId=a7511e51683dd, content=<a href='/topic/show?id=2c2c1553e56' target=_blank style='color:#2F92EE;'>#RNAi#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=15537, encryptionId=2c2c1553e56, topicName=RNAi)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=d9d436366397, createdName=yinxm8315, createdTime=Sun Apr 04 12:20:29 CST 2021, time=2021-04-04, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1921064, encodeId=4a46192106444, content=<a href='/topic/show?id=921b5e08167' target=_blank style='color:#2F92EE;'>#支持治疗#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=29, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=57081, encryptionId=921b5e08167, topicName=支持治疗)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=640c454, createdName=qblt, createdTime=Sat Aug 29 00:20:29 CST 2020, time=2020-08-29, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=803539, encodeId=21d5803539e9, content=原发性高草酸, beContent=null, objectType=article, channel=null, level=null, likeNumber=87, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20200720/956eb01f4b594ee783debae7a44d9096/e569cb8381764328b493c23491d86c27.jpg, createdBy=2cbc5401622, createdName=老婆子, createdTime=Mon Jul 20 20:20:22 CST 2020, time=2020-07-20, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=803216, encodeId=dfad803216c9, content=厉害, beContent=null, objectType=article, channel=null, level=null, likeNumber=89, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=27d45175397, createdName=146e617dm26暂无昵称, createdTime=Sat Jul 18 21:27:37 CST 2020, time=2020-07-18, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1536870, encodeId=7d1215368e016, content=<a href='/topic/show?id=7cad1554076' target=_blank style='color:#2F92EE;'>#RNAi疗法Lumasiran#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=28, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=15540, encryptionId=7cad1554076, topicName=RNAi疗法Lumasiran)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=fe1712732896, createdName=fzwish20004, createdTime=Sat Jul 18 10:20:29 CST 2020, time=2020-07-18, status=1, ipAttribution=)]
  3. [GetPortalCommentsPageByObjectIdResponse(id=1846915, encodeId=330e1846915ad, content=<a href='/topic/show?id=d3a33e0306d' target=_blank style='color:#2F92EE;'>#原发性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=26, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=37030, encryptionId=d3a33e0306d, topicName=原发性)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=8e4c53, createdName=jiyangfei, createdTime=Fri Jan 29 20:20:29 CST 2021, time=2021-01-29, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1648488, encodeId=4d9316484884b, content=<a href='/topic/show?id=c3b68e084ff' target=_blank style='color:#2F92EE;'>#英国MHRA#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=28, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=87084, encryptionId=c3b68e084ff, topicName=英国MHRA)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=e32f23584568, createdName=minzju5060, createdTime=Fri Mar 19 02:20:29 CST 2021, time=2021-03-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1751683, encodeId=a7511e51683dd, content=<a href='/topic/show?id=2c2c1553e56' target=_blank style='color:#2F92EE;'>#RNAi#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=15537, encryptionId=2c2c1553e56, topicName=RNAi)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=d9d436366397, createdName=yinxm8315, createdTime=Sun Apr 04 12:20:29 CST 2021, time=2021-04-04, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1921064, encodeId=4a46192106444, content=<a href='/topic/show?id=921b5e08167' target=_blank style='color:#2F92EE;'>#支持治疗#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=29, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=57081, encryptionId=921b5e08167, topicName=支持治疗)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=640c454, createdName=qblt, createdTime=Sat Aug 29 00:20:29 CST 2020, time=2020-08-29, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=803539, encodeId=21d5803539e9, content=原发性高草酸, beContent=null, objectType=article, channel=null, level=null, likeNumber=87, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20200720/956eb01f4b594ee783debae7a44d9096/e569cb8381764328b493c23491d86c27.jpg, createdBy=2cbc5401622, createdName=老婆子, createdTime=Mon Jul 20 20:20:22 CST 2020, time=2020-07-20, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=803216, encodeId=dfad803216c9, content=厉害, beContent=null, objectType=article, channel=null, level=null, likeNumber=89, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=27d45175397, createdName=146e617dm26暂无昵称, createdTime=Sat Jul 18 21:27:37 CST 2020, time=2020-07-18, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1536870, encodeId=7d1215368e016, content=<a href='/topic/show?id=7cad1554076' target=_blank style='color:#2F92EE;'>#RNAi疗法Lumasiran#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=28, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=15540, encryptionId=7cad1554076, topicName=RNAi疗法Lumasiran)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=fe1712732896, createdName=fzwish20004, createdTime=Sat Jul 18 10:20:29 CST 2020, time=2020-07-18, status=1, ipAttribution=)]
    2021-04-04 yinxm8315
  4. [GetPortalCommentsPageByObjectIdResponse(id=1846915, encodeId=330e1846915ad, content=<a href='/topic/show?id=d3a33e0306d' target=_blank style='color:#2F92EE;'>#原发性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=26, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=37030, encryptionId=d3a33e0306d, topicName=原发性)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=8e4c53, createdName=jiyangfei, createdTime=Fri Jan 29 20:20:29 CST 2021, time=2021-01-29, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1648488, encodeId=4d9316484884b, content=<a href='/topic/show?id=c3b68e084ff' target=_blank style='color:#2F92EE;'>#英国MHRA#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=28, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=87084, encryptionId=c3b68e084ff, topicName=英国MHRA)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=e32f23584568, createdName=minzju5060, createdTime=Fri Mar 19 02:20:29 CST 2021, time=2021-03-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1751683, encodeId=a7511e51683dd, content=<a href='/topic/show?id=2c2c1553e56' target=_blank style='color:#2F92EE;'>#RNAi#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=15537, encryptionId=2c2c1553e56, topicName=RNAi)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=d9d436366397, createdName=yinxm8315, createdTime=Sun Apr 04 12:20:29 CST 2021, time=2021-04-04, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1921064, encodeId=4a46192106444, content=<a href='/topic/show?id=921b5e08167' target=_blank style='color:#2F92EE;'>#支持治疗#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=29, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=57081, encryptionId=921b5e08167, topicName=支持治疗)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=640c454, createdName=qblt, createdTime=Sat Aug 29 00:20:29 CST 2020, time=2020-08-29, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=803539, encodeId=21d5803539e9, content=原发性高草酸, beContent=null, objectType=article, channel=null, level=null, likeNumber=87, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20200720/956eb01f4b594ee783debae7a44d9096/e569cb8381764328b493c23491d86c27.jpg, createdBy=2cbc5401622, createdName=老婆子, createdTime=Mon Jul 20 20:20:22 CST 2020, time=2020-07-20, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=803216, encodeId=dfad803216c9, content=厉害, beContent=null, objectType=article, channel=null, level=null, likeNumber=89, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=27d45175397, createdName=146e617dm26暂无昵称, createdTime=Sat Jul 18 21:27:37 CST 2020, time=2020-07-18, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1536870, encodeId=7d1215368e016, content=<a href='/topic/show?id=7cad1554076' target=_blank style='color:#2F92EE;'>#RNAi疗法Lumasiran#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=28, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=15540, encryptionId=7cad1554076, topicName=RNAi疗法Lumasiran)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=fe1712732896, createdName=fzwish20004, createdTime=Sat Jul 18 10:20:29 CST 2020, time=2020-07-18, status=1, ipAttribution=)]
    2020-08-29 qblt
  5. [GetPortalCommentsPageByObjectIdResponse(id=1846915, encodeId=330e1846915ad, content=<a href='/topic/show?id=d3a33e0306d' target=_blank style='color:#2F92EE;'>#原发性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=26, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=37030, encryptionId=d3a33e0306d, topicName=原发性)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=8e4c53, createdName=jiyangfei, createdTime=Fri Jan 29 20:20:29 CST 2021, time=2021-01-29, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1648488, encodeId=4d9316484884b, content=<a href='/topic/show?id=c3b68e084ff' target=_blank style='color:#2F92EE;'>#英国MHRA#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=28, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=87084, encryptionId=c3b68e084ff, topicName=英国MHRA)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=e32f23584568, createdName=minzju5060, createdTime=Fri Mar 19 02:20:29 CST 2021, time=2021-03-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1751683, encodeId=a7511e51683dd, content=<a href='/topic/show?id=2c2c1553e56' target=_blank style='color:#2F92EE;'>#RNAi#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=15537, encryptionId=2c2c1553e56, topicName=RNAi)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=d9d436366397, createdName=yinxm8315, createdTime=Sun Apr 04 12:20:29 CST 2021, time=2021-04-04, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1921064, encodeId=4a46192106444, content=<a href='/topic/show?id=921b5e08167' target=_blank style='color:#2F92EE;'>#支持治疗#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=29, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=57081, encryptionId=921b5e08167, topicName=支持治疗)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=640c454, createdName=qblt, createdTime=Sat Aug 29 00:20:29 CST 2020, time=2020-08-29, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=803539, encodeId=21d5803539e9, content=原发性高草酸, beContent=null, objectType=article, channel=null, level=null, likeNumber=87, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20200720/956eb01f4b594ee783debae7a44d9096/e569cb8381764328b493c23491d86c27.jpg, createdBy=2cbc5401622, createdName=老婆子, createdTime=Mon Jul 20 20:20:22 CST 2020, time=2020-07-20, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=803216, encodeId=dfad803216c9, content=厉害, beContent=null, objectType=article, channel=null, level=null, likeNumber=89, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=27d45175397, createdName=146e617dm26暂无昵称, createdTime=Sat Jul 18 21:27:37 CST 2020, time=2020-07-18, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1536870, encodeId=7d1215368e016, content=<a href='/topic/show?id=7cad1554076' target=_blank style='color:#2F92EE;'>#RNAi疗法Lumasiran#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=28, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=15540, encryptionId=7cad1554076, topicName=RNAi疗法Lumasiran)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=fe1712732896, createdName=fzwish20004, createdTime=Sat Jul 18 10:20:29 CST 2020, time=2020-07-18, status=1, ipAttribution=)]
    2020-07-20 老婆子

    原发性高草酸

    0

  6. [GetPortalCommentsPageByObjectIdResponse(id=1846915, encodeId=330e1846915ad, content=<a href='/topic/show?id=d3a33e0306d' target=_blank style='color:#2F92EE;'>#原发性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=26, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=37030, encryptionId=d3a33e0306d, topicName=原发性)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=8e4c53, createdName=jiyangfei, createdTime=Fri Jan 29 20:20:29 CST 2021, time=2021-01-29, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1648488, encodeId=4d9316484884b, content=<a href='/topic/show?id=c3b68e084ff' target=_blank style='color:#2F92EE;'>#英国MHRA#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=28, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=87084, encryptionId=c3b68e084ff, topicName=英国MHRA)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=e32f23584568, createdName=minzju5060, createdTime=Fri Mar 19 02:20:29 CST 2021, time=2021-03-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1751683, encodeId=a7511e51683dd, content=<a href='/topic/show?id=2c2c1553e56' target=_blank style='color:#2F92EE;'>#RNAi#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=15537, encryptionId=2c2c1553e56, topicName=RNAi)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=d9d436366397, createdName=yinxm8315, createdTime=Sun Apr 04 12:20:29 CST 2021, time=2021-04-04, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1921064, encodeId=4a46192106444, content=<a href='/topic/show?id=921b5e08167' target=_blank style='color:#2F92EE;'>#支持治疗#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=29, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=57081, encryptionId=921b5e08167, topicName=支持治疗)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=640c454, createdName=qblt, createdTime=Sat Aug 29 00:20:29 CST 2020, time=2020-08-29, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=803539, encodeId=21d5803539e9, content=原发性高草酸, beContent=null, objectType=article, channel=null, level=null, likeNumber=87, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20200720/956eb01f4b594ee783debae7a44d9096/e569cb8381764328b493c23491d86c27.jpg, createdBy=2cbc5401622, createdName=老婆子, createdTime=Mon Jul 20 20:20:22 CST 2020, time=2020-07-20, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=803216, encodeId=dfad803216c9, content=厉害, beContent=null, objectType=article, channel=null, level=null, likeNumber=89, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=27d45175397, createdName=146e617dm26暂无昵称, createdTime=Sat Jul 18 21:27:37 CST 2020, time=2020-07-18, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1536870, encodeId=7d1215368e016, content=<a href='/topic/show?id=7cad1554076' target=_blank style='color:#2F92EE;'>#RNAi疗法Lumasiran#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=28, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=15540, encryptionId=7cad1554076, topicName=RNAi疗法Lumasiran)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=fe1712732896, createdName=fzwish20004, createdTime=Sat Jul 18 10:20:29 CST 2020, time=2020-07-18, status=1, ipAttribution=)]
    2020-07-18 146e617dm26暂无昵称

    厉害

    0

  7. [GetPortalCommentsPageByObjectIdResponse(id=1846915, encodeId=330e1846915ad, content=<a href='/topic/show?id=d3a33e0306d' target=_blank style='color:#2F92EE;'>#原发性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=26, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=37030, encryptionId=d3a33e0306d, topicName=原发性)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=8e4c53, createdName=jiyangfei, createdTime=Fri Jan 29 20:20:29 CST 2021, time=2021-01-29, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1648488, encodeId=4d9316484884b, content=<a href='/topic/show?id=c3b68e084ff' target=_blank style='color:#2F92EE;'>#英国MHRA#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=28, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=87084, encryptionId=c3b68e084ff, topicName=英国MHRA)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=e32f23584568, createdName=minzju5060, createdTime=Fri Mar 19 02:20:29 CST 2021, time=2021-03-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1751683, encodeId=a7511e51683dd, content=<a href='/topic/show?id=2c2c1553e56' target=_blank style='color:#2F92EE;'>#RNAi#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=15537, encryptionId=2c2c1553e56, topicName=RNAi)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=d9d436366397, createdName=yinxm8315, createdTime=Sun Apr 04 12:20:29 CST 2021, time=2021-04-04, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1921064, encodeId=4a46192106444, content=<a href='/topic/show?id=921b5e08167' target=_blank style='color:#2F92EE;'>#支持治疗#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=29, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=57081, encryptionId=921b5e08167, topicName=支持治疗)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=640c454, createdName=qblt, createdTime=Sat Aug 29 00:20:29 CST 2020, time=2020-08-29, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=803539, encodeId=21d5803539e9, content=原发性高草酸, beContent=null, objectType=article, channel=null, level=null, likeNumber=87, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20200720/956eb01f4b594ee783debae7a44d9096/e569cb8381764328b493c23491d86c27.jpg, createdBy=2cbc5401622, createdName=老婆子, createdTime=Mon Jul 20 20:20:22 CST 2020, time=2020-07-20, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=803216, encodeId=dfad803216c9, content=厉害, beContent=null, objectType=article, channel=null, level=null, likeNumber=89, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=27d45175397, createdName=146e617dm26暂无昵称, createdTime=Sat Jul 18 21:27:37 CST 2020, time=2020-07-18, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1536870, encodeId=7d1215368e016, content=<a href='/topic/show?id=7cad1554076' target=_blank style='color:#2F92EE;'>#RNAi疗法Lumasiran#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=28, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=15540, encryptionId=7cad1554076, topicName=RNAi疗法Lumasiran)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=fe1712732896, createdName=fzwish20004, createdTime=Sat Jul 18 10:20:29 CST 2020, time=2020-07-18, status=1, ipAttribution=)]

相关资讯

ERA-EDTA 2020:RNAi疗法公司Alnylam再发力!Lumasiran治疗1型原发性高草酸尿症III期临床成功

Lumasiran达到ILLUMINATE-A的主要终点,相对于安慰剂而言,草酸尿的平均减少量为53.5%

Alynlam的RNAi药物lumasiran治疗原发性高草酸尿症,完成向欧盟和美国的申请

如果获得批准,lumasiran将成为1型原发性高草酸尿症(PH1)患者的首个治疗药物。

ERA-EDTA 2020:重磅!RNAi疗法Lumasiran治疗高草酸尿症,6个月患者恢复到正常水平

lumasiran治疗组,84%的患者草酸尿排泄达到了正常化或接近正常化的水平,而安慰剂组没有一人实现该目标。

FDA为Alnylam的RNAi疗法Lumasiran授予优先审查,用于治疗1型原发性高草酸尿症

Lumasiran是靶向羟基酸氧化酶1(HAO1)、可按季度皮下给药的的RNAi治疗药物。