MS1819-SD结合PERT治疗严重外分泌胰腺功能不全的囊性纤维化患者

2019-07-09 Allan MedSci原创

AzurRx是一家专门开发胃肠疾病疗法的生物技术公司,近日宣布,已启动一项II期临床试验,以评估MS1819-SD结合标准猪酶替代疗法(PERT)治疗患有严重外分泌胰腺功能不全(EPI)的囊性纤维化(CF)患者,这些患者尽管服用了大剂量的PERT,仍继续出现脂肪吸收不良的临床症状。

AzurRx是一家专门开发肠疾病疗法的生物技术公司,近日宣布,已启动一项II临床试验,以评估MS1819-SD结合标准猪酶替代疗法(PERT)治疗患有严重外分泌胰腺功能不全(EPI)的囊性纤维化(CF)患者,这些患者尽管服用了大剂量的PERT,仍继续出现脂肪吸收不良的临床症状。

理想情况下,稳定的每日剂量的PERT将使CF患者能够正常进食并最大限度地减少令人不快的肠道症状。然而,在实践中,大量CF患者不能通过PERT实现正常的脂肪吸收。在CF患者中实现最佳营养状态(包括正常脂肪吸收水平)对于维持更好的肺功能、身体表现和延长存活率是至关重要的。


原始出处:

http://www.firstwordpharma.com/node/1651946#axzz5syvQI0kQ

本文系梅斯医学(MedSci)原创编译整理,转载需授权!

评论区 (4)
#插入话题
  1. [GetPortalCommentsPageByObjectIdResponse(id=2006170, encodeId=85a220061e0aa, content=<a href='/topic/show?id=a5c54038142' target=_blank style='color:#2F92EE;'>#囊性纤维化患者#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=111, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=40381, encryptionId=a5c54038142, topicName=囊性纤维化患者)], attachment=null, authenticateStatus=null, createdAvatar=https://thirdqq.qlogo.cn/qqapp/1103841572/17F844FBE270B8196CB4C53EC9493C57/100, createdBy=9d712500079, createdName=ms5906905816781247, createdTime=Tue Apr 28 11:40:00 CST 2020, time=2020-04-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1728044, encodeId=f6651e2804444, content=<a href='/topic/show?id=986913980dd' target=_blank style='color:#2F92EE;'>#PE#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=85, 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 May 14 12:40:00 CST 2020, time=2020-05-14, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1682541, encodeId=921416825412b, content=<a href='/topic/show?id=d4d031e8074' target=_blank style='color:#2F92EE;'>#分泌#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=100, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=31780, encryptionId=d4d031e8074, topicName=分泌)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=0da227938338, createdName=gwc392, createdTime=Fri Jan 10 04:40:00 CST 2020, time=2020-01-10, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1675906, encodeId=4c0216e59069c, content=<a href='/topic/show?id=7395403e580' target=_blank style='color:#2F92EE;'>#囊性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=116, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=40375, encryptionId=7395403e580, topicName=囊性)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=926927300775, createdName=wolongzxh, createdTime=Sun Aug 25 12:40:00 CST 2019, time=2019-08-25, status=1, ipAttribution=)]
  2. [GetPortalCommentsPageByObjectIdResponse(id=2006170, encodeId=85a220061e0aa, content=<a href='/topic/show?id=a5c54038142' target=_blank style='color:#2F92EE;'>#囊性纤维化患者#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=111, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=40381, encryptionId=a5c54038142, topicName=囊性纤维化患者)], attachment=null, authenticateStatus=null, createdAvatar=https://thirdqq.qlogo.cn/qqapp/1103841572/17F844FBE270B8196CB4C53EC9493C57/100, createdBy=9d712500079, createdName=ms5906905816781247, createdTime=Tue Apr 28 11:40:00 CST 2020, time=2020-04-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1728044, encodeId=f6651e2804444, content=<a href='/topic/show?id=986913980dd' target=_blank style='color:#2F92EE;'>#PE#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=85, 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 May 14 12:40:00 CST 2020, time=2020-05-14, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1682541, encodeId=921416825412b, content=<a href='/topic/show?id=d4d031e8074' target=_blank style='color:#2F92EE;'>#分泌#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=100, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=31780, encryptionId=d4d031e8074, topicName=分泌)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=0da227938338, createdName=gwc392, createdTime=Fri Jan 10 04:40:00 CST 2020, time=2020-01-10, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1675906, encodeId=4c0216e59069c, content=<a href='/topic/show?id=7395403e580' target=_blank style='color:#2F92EE;'>#囊性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=116, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=40375, encryptionId=7395403e580, topicName=囊性)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=926927300775, createdName=wolongzxh, createdTime=Sun Aug 25 12:40:00 CST 2019, time=2019-08-25, status=1, ipAttribution=)]
    2020-05-14 feather89
  3. [GetPortalCommentsPageByObjectIdResponse(id=2006170, encodeId=85a220061e0aa, content=<a href='/topic/show?id=a5c54038142' target=_blank style='color:#2F92EE;'>#囊性纤维化患者#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=111, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=40381, encryptionId=a5c54038142, topicName=囊性纤维化患者)], attachment=null, authenticateStatus=null, createdAvatar=https://thirdqq.qlogo.cn/qqapp/1103841572/17F844FBE270B8196CB4C53EC9493C57/100, createdBy=9d712500079, createdName=ms5906905816781247, createdTime=Tue Apr 28 11:40:00 CST 2020, time=2020-04-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1728044, encodeId=f6651e2804444, content=<a href='/topic/show?id=986913980dd' target=_blank style='color:#2F92EE;'>#PE#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=85, 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 May 14 12:40:00 CST 2020, time=2020-05-14, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1682541, encodeId=921416825412b, content=<a href='/topic/show?id=d4d031e8074' target=_blank style='color:#2F92EE;'>#分泌#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=100, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=31780, encryptionId=d4d031e8074, topicName=分泌)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=0da227938338, createdName=gwc392, createdTime=Fri Jan 10 04:40:00 CST 2020, time=2020-01-10, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1675906, encodeId=4c0216e59069c, content=<a href='/topic/show?id=7395403e580' target=_blank style='color:#2F92EE;'>#囊性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=116, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=40375, encryptionId=7395403e580, topicName=囊性)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=926927300775, createdName=wolongzxh, createdTime=Sun Aug 25 12:40:00 CST 2019, time=2019-08-25, status=1, ipAttribution=)]
    2020-01-10 gwc392
  4. [GetPortalCommentsPageByObjectIdResponse(id=2006170, encodeId=85a220061e0aa, content=<a href='/topic/show?id=a5c54038142' target=_blank style='color:#2F92EE;'>#囊性纤维化患者#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=111, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=40381, encryptionId=a5c54038142, topicName=囊性纤维化患者)], attachment=null, authenticateStatus=null, createdAvatar=https://thirdqq.qlogo.cn/qqapp/1103841572/17F844FBE270B8196CB4C53EC9493C57/100, createdBy=9d712500079, createdName=ms5906905816781247, createdTime=Tue Apr 28 11:40:00 CST 2020, time=2020-04-28, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1728044, encodeId=f6651e2804444, content=<a href='/topic/show?id=986913980dd' target=_blank style='color:#2F92EE;'>#PE#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=85, 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 May 14 12:40:00 CST 2020, time=2020-05-14, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1682541, encodeId=921416825412b, content=<a href='/topic/show?id=d4d031e8074' target=_blank style='color:#2F92EE;'>#分泌#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=100, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=31780, encryptionId=d4d031e8074, topicName=分泌)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=0da227938338, createdName=gwc392, createdTime=Fri Jan 10 04:40:00 CST 2020, time=2020-01-10, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1675906, encodeId=4c0216e59069c, content=<a href='/topic/show?id=7395403e580' target=_blank style='color:#2F92EE;'>#囊性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=116, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=40375, encryptionId=7395403e580, topicName=囊性)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=926927300775, createdName=wolongzxh, createdTime=Sun Aug 25 12:40:00 CST 2019, time=2019-08-25, status=1, ipAttribution=)]
    2019-08-25 wolongzxh

相关资讯

Vertex公司的SYMDEKO获澳大利亚批准,用于治疗12岁以上CFTR基因突变的囊性纤维化患者

波士顿Vertex制药股份有限公司今天宣布,澳大利亚治疗产品管理局(TGA)已批准将SYMDEKO(tezacaftor / ivacaftor和ivacaftor)上市,用于F508del突变或囊性纤维化跨膜传导调节因子(CFTR)基因突变的12岁以上囊性纤维化(CF)患者的治疗。

Vertex新的三联组合治疗囊性纤维化,3期临床大获成功

自2012年推出Kalydeco以来,总部位于马萨诸塞州波士顿的Vertex公司在囊性纤维化(CF)治疗方面一直处于领先地位。全球CF患者约75000人,其目前的Kalydeco,Orkambi和Symkevi组合只能治疗这一总数的一半,Vertex公司的最终目标是治疗所有的CF患者。

Vertex的VX-445三联组合方案治疗囊性纤维化的III期研究达到主要终点

Vertex制药公司在本周三(2019年3月6日)宣布,研究性新药VX-445与ivacaftor和tezacaftor结合的三联方案治疗囊性纤维化(CF)患者的两项III期研究均符合其主要终点。结果显示,与安慰剂相比,三联疗法与肺功能的改善显著相关。

NATURE:小分子离子通道增加囊性纤维化气道上皮细胞的宿主防御

最近,研究人员发现,顶端添加两性霉素B,一种形成非选择性离子通道的小分子,可以恢复HCO3-分泌,并增加来自囊性纤维化患者的气道上皮细胞的气道表面液体pH。

2019 CFF共识指南:囊性纤维化患者肺移植推荐

2019年3月,囊性纤维化基金会(CFF)发布了囊性纤维化患者肺移植推荐指南,本文的主要目的是为囊性纤维化患者转诊进行肺移植提供推荐意见,全文共提出了21条建议声明。

囊性纤维化候选药物SNSP113获得欧盟孤儿药物认定

Synspira Therapeutics近日宣布,欧洲药品管理局(EMA)孤儿药品委员会(COMP)已授予该公司的SNSP113以孤儿药物认定。