欧盟批准Vertex囊性纤维化药物Kalydeco用于8种非G551D门控突变

2014-08-04 佚名 生物谷

-福泰制药(Vertex)7月31日宣布,药物Kalydeco(ivacaftor)获欧盟委员会(EC)批准,用于囊性纤维化跨膜电导调节因子(CFTR)基因中存在8种非G551D门控突变(non-G551D gating mutations)之一的6岁及以上囊性纤维化患者的治疗。此次获批,是基于二部分、随机、双盲、安慰剂对照III期研究的第一部分数据,该研究在39例携带非G551D门控突变的6岁及

-福泰制药(Vertex)7月31日宣布,药物Kalydeco(ivacaftor)获欧盟委员会(EC)批准,用于囊性纤维化跨膜电导调节因子(CFTR)基因中存在8种非G551D门控突变(non-G551D gating mutations)之一的6岁及以上囊性纤维化患者的治疗。

此次获批,是基于二部分、随机、双盲、安慰剂对照III期研究的第一部分数据,该研究在39例携带非G551D门控突变的6岁及以上囊性纤维化(CF)患者中开展。第一部分数据表明,Kalydeco使肺功能(FEV1)、汗液氯化物、身体质量指数、CFQ-R得分均取得了统计学意义的显著改善。该项研究的第二部分数据已于2014年6月提交至欧洲囊性纤维化协会会议,数据表明,Kalydeco在研究的第一部分中所取得的改善,在整个治疗24周中均能够维持。该项研究中的安全性与在G551D门控突变患者中开展的III期研究一致。

Kalydeco是首个靶向囊性纤维化(CF)根本病因的药物,可使G551D突变患者体内缺陷性CFTR蛋白发挥正常功能。G551D突变是一种最常见的门控突变,该突变损害了ATP介导的通道调节。

此次获批的8种非G551D门控突变包括:G178R,S549N,S549R,G551S,G1244E,S1251N,S1255P和G1349D。在欧洲,约有250例患者携带这8种非G551D门控突变。

关于Kalydeco:

Kalydeco于2012年首次获FDA及EMA批准,用于治疗CFTR基因存在至少单拷贝G551D突变的6岁及以上囊性纤维化(CF)患者。此外,FDA于2014年6月批准Kalydeco用于携带9种非G551D门控突变中任意一种突变的6岁及以上CF患者,包括:G178R,S549N,S549R,G551S,G1244E,S1251N,S1255P,G1349D或G970R。

关于囊性纤维化(CF):

囊性纤维化(CF)是由囊性纤维化跨膜电导调节因子(CFTR)基因突变导致CFTR蛋白功能缺陷或缺失所致的罕见遗传性疾病,该病困扰着全球约7万人。CFTR蛋白通常调节细胞膜的离子运输,基因突变能导致蛋白产物功能的破坏或丧失。当细胞膜离子运输被中断,某些器官粘液涂层的粘度将变稠。该病的一个主要特征是呼吸道积聚厚厚的粘液,导致呼吸困难及反复感染。


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 (5)
#插入话题
  1. [GetPortalCommentsPageByObjectIdResponse(id=1675812, encodeId=8e4b16e5812de, content=<a href='/topic/show?id=7395403e580' target=_blank style='color:#2F92EE;'>#囊性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=21, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=40375, encryptionId=7395403e580, topicName=囊性)], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/aLGWoFXAyMbIu3qymFOyheQLjPSX3OUs5GmkyBlcCOwTPIeq3why9NGibxxUqYo6hcx8qZLHZFgNPnBK1yzWeOFpyg2OnWOt0/0, createdBy=fa4716, createdName=仁心济世, createdTime=Thu Jan 08 18:10:00 CST 2015, time=2015-01-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1973605, encodeId=0f7719e360509, content=<a href='/topic/show?id=735a40386ed' target=_blank style='color:#2F92EE;'>#囊性纤维化药物#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=35, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=40386, encryptionId=735a40386ed, topicName=囊性纤维化药物)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=38232500109, createdName=1249898am15暂无昵称, createdTime=Mon Nov 24 09:10:00 CST 2014, time=2014-11-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1417932, encodeId=b761141e932cf, content=<a href='/topic/show?id=4b711839233' target=_blank style='color:#2F92EE;'>#Vertex#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=32, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=18392, encryptionId=4b711839233, topicName=Vertex)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=4e443309191, createdName=liuli5080, createdTime=Wed Aug 06 00:10:00 CST 2014, time=2014-08-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1589939, encodeId=bb4515899399a, content=<a href='/topic/show?id=2d1c63118e9' target=_blank style='color:#2F92EE;'>#欧盟批准#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=21, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=63118, encryptionId=2d1c63118e9, topicName=欧盟批准)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f0d617780998, createdName=chenwq14, createdTime=Wed Aug 06 00:10:00 CST 2014, time=2014-08-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1621463, encodeId=d038162146317, content=<a href='/topic/show?id=559c104056d' target=_blank style='color:#2F92EE;'>#Kalydeco#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=23, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=10405, encryptionId=559c104056d, topicName=Kalydeco)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=d49420270114, createdName=windight, createdTime=Wed Aug 06 00:10:00 CST 2014, time=2014-08-06, status=1, ipAttribution=)]
  2. [GetPortalCommentsPageByObjectIdResponse(id=1675812, encodeId=8e4b16e5812de, content=<a href='/topic/show?id=7395403e580' target=_blank style='color:#2F92EE;'>#囊性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=21, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=40375, encryptionId=7395403e580, topicName=囊性)], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/aLGWoFXAyMbIu3qymFOyheQLjPSX3OUs5GmkyBlcCOwTPIeq3why9NGibxxUqYo6hcx8qZLHZFgNPnBK1yzWeOFpyg2OnWOt0/0, createdBy=fa4716, createdName=仁心济世, createdTime=Thu Jan 08 18:10:00 CST 2015, time=2015-01-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1973605, encodeId=0f7719e360509, content=<a href='/topic/show?id=735a40386ed' target=_blank style='color:#2F92EE;'>#囊性纤维化药物#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=35, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=40386, encryptionId=735a40386ed, topicName=囊性纤维化药物)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=38232500109, createdName=1249898am15暂无昵称, createdTime=Mon Nov 24 09:10:00 CST 2014, time=2014-11-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1417932, encodeId=b761141e932cf, content=<a href='/topic/show?id=4b711839233' target=_blank style='color:#2F92EE;'>#Vertex#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=32, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=18392, encryptionId=4b711839233, topicName=Vertex)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=4e443309191, createdName=liuli5080, createdTime=Wed Aug 06 00:10:00 CST 2014, time=2014-08-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1589939, encodeId=bb4515899399a, content=<a href='/topic/show?id=2d1c63118e9' target=_blank style='color:#2F92EE;'>#欧盟批准#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=21, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=63118, encryptionId=2d1c63118e9, topicName=欧盟批准)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f0d617780998, createdName=chenwq14, createdTime=Wed Aug 06 00:10:00 CST 2014, time=2014-08-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1621463, encodeId=d038162146317, content=<a href='/topic/show?id=559c104056d' target=_blank style='color:#2F92EE;'>#Kalydeco#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=23, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=10405, encryptionId=559c104056d, topicName=Kalydeco)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=d49420270114, createdName=windight, createdTime=Wed Aug 06 00:10:00 CST 2014, time=2014-08-06, status=1, ipAttribution=)]
  3. [GetPortalCommentsPageByObjectIdResponse(id=1675812, encodeId=8e4b16e5812de, content=<a href='/topic/show?id=7395403e580' target=_blank style='color:#2F92EE;'>#囊性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=21, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=40375, encryptionId=7395403e580, topicName=囊性)], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/aLGWoFXAyMbIu3qymFOyheQLjPSX3OUs5GmkyBlcCOwTPIeq3why9NGibxxUqYo6hcx8qZLHZFgNPnBK1yzWeOFpyg2OnWOt0/0, createdBy=fa4716, createdName=仁心济世, createdTime=Thu Jan 08 18:10:00 CST 2015, time=2015-01-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1973605, encodeId=0f7719e360509, content=<a href='/topic/show?id=735a40386ed' target=_blank style='color:#2F92EE;'>#囊性纤维化药物#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=35, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=40386, encryptionId=735a40386ed, topicName=囊性纤维化药物)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=38232500109, createdName=1249898am15暂无昵称, createdTime=Mon Nov 24 09:10:00 CST 2014, time=2014-11-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1417932, encodeId=b761141e932cf, content=<a href='/topic/show?id=4b711839233' target=_blank style='color:#2F92EE;'>#Vertex#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=32, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=18392, encryptionId=4b711839233, topicName=Vertex)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=4e443309191, createdName=liuli5080, createdTime=Wed Aug 06 00:10:00 CST 2014, time=2014-08-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1589939, encodeId=bb4515899399a, content=<a href='/topic/show?id=2d1c63118e9' target=_blank style='color:#2F92EE;'>#欧盟批准#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=21, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=63118, encryptionId=2d1c63118e9, topicName=欧盟批准)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f0d617780998, createdName=chenwq14, createdTime=Wed Aug 06 00:10:00 CST 2014, time=2014-08-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1621463, encodeId=d038162146317, content=<a href='/topic/show?id=559c104056d' target=_blank style='color:#2F92EE;'>#Kalydeco#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=23, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=10405, encryptionId=559c104056d, topicName=Kalydeco)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=d49420270114, createdName=windight, createdTime=Wed Aug 06 00:10:00 CST 2014, time=2014-08-06, status=1, ipAttribution=)]
    2014-08-06 liuli5080
  4. [GetPortalCommentsPageByObjectIdResponse(id=1675812, encodeId=8e4b16e5812de, content=<a href='/topic/show?id=7395403e580' target=_blank style='color:#2F92EE;'>#囊性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=21, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=40375, encryptionId=7395403e580, topicName=囊性)], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/aLGWoFXAyMbIu3qymFOyheQLjPSX3OUs5GmkyBlcCOwTPIeq3why9NGibxxUqYo6hcx8qZLHZFgNPnBK1yzWeOFpyg2OnWOt0/0, createdBy=fa4716, createdName=仁心济世, createdTime=Thu Jan 08 18:10:00 CST 2015, time=2015-01-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1973605, encodeId=0f7719e360509, content=<a href='/topic/show?id=735a40386ed' target=_blank style='color:#2F92EE;'>#囊性纤维化药物#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=35, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=40386, encryptionId=735a40386ed, topicName=囊性纤维化药物)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=38232500109, createdName=1249898am15暂无昵称, createdTime=Mon Nov 24 09:10:00 CST 2014, time=2014-11-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1417932, encodeId=b761141e932cf, content=<a href='/topic/show?id=4b711839233' target=_blank style='color:#2F92EE;'>#Vertex#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=32, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=18392, encryptionId=4b711839233, topicName=Vertex)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=4e443309191, createdName=liuli5080, createdTime=Wed Aug 06 00:10:00 CST 2014, time=2014-08-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1589939, encodeId=bb4515899399a, content=<a href='/topic/show?id=2d1c63118e9' target=_blank style='color:#2F92EE;'>#欧盟批准#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=21, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=63118, encryptionId=2d1c63118e9, topicName=欧盟批准)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f0d617780998, createdName=chenwq14, createdTime=Wed Aug 06 00:10:00 CST 2014, time=2014-08-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1621463, encodeId=d038162146317, content=<a href='/topic/show?id=559c104056d' target=_blank style='color:#2F92EE;'>#Kalydeco#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=23, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=10405, encryptionId=559c104056d, topicName=Kalydeco)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=d49420270114, createdName=windight, createdTime=Wed Aug 06 00:10:00 CST 2014, time=2014-08-06, status=1, ipAttribution=)]
  5. [GetPortalCommentsPageByObjectIdResponse(id=1675812, encodeId=8e4b16e5812de, content=<a href='/topic/show?id=7395403e580' target=_blank style='color:#2F92EE;'>#囊性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=21, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=40375, encryptionId=7395403e580, topicName=囊性)], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/aLGWoFXAyMbIu3qymFOyheQLjPSX3OUs5GmkyBlcCOwTPIeq3why9NGibxxUqYo6hcx8qZLHZFgNPnBK1yzWeOFpyg2OnWOt0/0, createdBy=fa4716, createdName=仁心济世, createdTime=Thu Jan 08 18:10:00 CST 2015, time=2015-01-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1973605, encodeId=0f7719e360509, content=<a href='/topic/show?id=735a40386ed' target=_blank style='color:#2F92EE;'>#囊性纤维化药物#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=35, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=40386, encryptionId=735a40386ed, topicName=囊性纤维化药物)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=38232500109, createdName=1249898am15暂无昵称, createdTime=Mon Nov 24 09:10:00 CST 2014, time=2014-11-24, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1417932, encodeId=b761141e932cf, content=<a href='/topic/show?id=4b711839233' target=_blank style='color:#2F92EE;'>#Vertex#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=32, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=18392, encryptionId=4b711839233, topicName=Vertex)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=4e443309191, createdName=liuli5080, createdTime=Wed Aug 06 00:10:00 CST 2014, time=2014-08-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1589939, encodeId=bb4515899399a, content=<a href='/topic/show?id=2d1c63118e9' target=_blank style='color:#2F92EE;'>#欧盟批准#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=21, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=63118, encryptionId=2d1c63118e9, topicName=欧盟批准)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f0d617780998, createdName=chenwq14, createdTime=Wed Aug 06 00:10:00 CST 2014, time=2014-08-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1621463, encodeId=d038162146317, content=<a href='/topic/show?id=559c104056d' target=_blank style='color:#2F92EE;'>#Kalydeco#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=23, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=10405, encryptionId=559c104056d, topicName=Kalydeco)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=d49420270114, createdName=windight, createdTime=Wed Aug 06 00:10:00 CST 2014, time=2014-08-06, status=1, ipAttribution=)]
    2014-08-06 windight

相关资讯

Nat Commun:研究证实CFTR蛋白参与调控胰岛素分泌

来自香港中文大学、四川大学等处的研究人员证实,在胰岛β细胞中囊性纤维化跨膜转运调节因子(cystic fibrosis transmembrane conductance regulator,CFTR)蛋白调控了葡萄糖诱导的电活动和胰岛素分泌。这一研究发现发表在7月15日的《自然通讯》(Nature Communications)杂志上。

文章的通讯作者是香港中文大学的陈小章(Hsiao Chang Chan)教授。陈小章教授一直从事人类生育相关的上皮细胞生物学研究,曾发表有关控制生育分子机制和不育成因等多个重要研究发现,为此获得过2次国家自然科学奖和2007年***成就奖。

糖尿病是由于胰岛素分泌不足(1型糖尿病)或胰岛素耐受(2型糖尿病)所引起的一种慢性代谢性疾病,全球约有300多万人受累于这一疾病。在2型糖尿病中,胰岛素抵抗之外还常伴有胰岛素分泌不足及胰岛β细胞受损。尽管人们通常将胰岛素不足视作是自身免疫损伤β细胞的结果,很高比例糖尿病患者却显示自身抗体阴性,对于这些患者出现

NEJM:CF患儿感染支气管肺炎的危险因素

  澳大利亚学者的一项研究显示,支气管肺泡灌洗(BAL)液体中的中性粒细胞弹性蛋白酶的活性与囊性纤维化(CF)患儿早期支气管肺炎相关。相关论文于2013年5月23日在线发表于《新英格兰医学杂志》(N Engl J Med)。    该研究纳入127例在新生儿筛查中被诊断为CF的婴儿,在孩子3个月,1岁和3岁时,分别进行胸部CT扫描和BAL。用纵向数据来决定危险因素与3个月到3岁支气管肺

阿仑膦酸可改善囊性纤维化患者骨密度

囊性纤维化的长期并发症包括骨质疏松和脆性骨折,但很少有资料显示有效的治疗策略,尤其在年轻患者中。我们调查了儿童、青少年和年轻囊性纤维化患者中有关低骨密度的治疗。意大利学者的一项研究显示,阿仑膦酸可改善囊性纤维化患者骨密度。相关研究近期发表于the Lancet杂志。 该多中心随机对照研究分两期。先从意大利10所囊性纤维化治疗中心纳入5-30岁的囊性纤维化且低骨密度患者。一期试验为12个

PLoS Gene:研究表明囊性纤维化是两种类型疾病

一项由美国匹兹堡大学医学院多中心团队完成的新研究提示:囊性纤维化(CF)其实是两种不同的疾病,一种影响多个器官包括肺,一种不影响肺。这项研究发表在 PLOS Genetics杂志上,揭示了与囊性纤维化相关基因变异中的9个可导致胰腺炎,鼻窦炎和男性不育症,但却不对肺部造成伤害。 CF患者继承分别来自父母各方的CFTR基因的一个严重突变拷贝,CFTR生成那些构成通道来转运氯化物分子进出细胞的蛋白质,

科学家在治疗囊性纤维化中获得重大突破

近日,女王大学科学家在治疗囊性纤维化中获得了重大的突破。女王大学的教授Stuart Elborn与来自美国和澳大利亚的同事领导了这项囊性纤维化新疗法的关键性研究。由Vertex (波士美国顿,公司)开发的组合药物治疗能改善肺功能,减少最常见类型囊性纤维化患者的住院治疗。 在两项药物ivacaftor和lumacaftor联合治疗的3期研究中,其中包括超过1,100名患者(以往研究已经探究iva

AAOS教程:细菌生物膜与假体周围感染

假体周围感染是人工关节置换术后的一种严重并发症,即使最终得到有效处理,其治疗期限也通常较长,同时患者也会经历长时期的关节功能丧失并且难以继续参与日常工作,使得总体治疗结果较差。 造成疗效较差的主要原因在于往往难以对假体周围感染早期作出准确的诊断,并由此给予正确的处理。对假体周围感染基本病理变化的理解有助于指导对其作出正确的诊断和处理。 目前针对包括假体周围感染在内各种感染的诊断