神经科

订阅

MedSci主页 / 所有栏目 / 神经科

FDA批准Emflaza治疗年轻DMD患者

2019-06-10 不详 网络

Emflaza杜兴氏肌肉营养不良症deflazacort
关注

PTC Therapeutics公司近日表示，FDA已将Emflaza（deflazacort）的适应症标签扩大到年龄在2岁到5岁之间的杜兴氏肌肉营养不良症（DMD）的患者。2017年，FDA批准了Emflaza用于5岁及以上DMD患者。

PTC Therapeutics公司近日表示，FDA已将Emflaza（deflazacort）的适应症标签扩大到年龄在2岁到5岁之间的杜兴氏肌肉营养不良症（DMD）的患者。2017年，FDA批准了Emflaza用于5岁及以上DMD患者。PTC Therapeutics首席执行官Stuart Peltz说：“对于所有DMD患者，Emflaza是唯一获得批准的治疗方法。我们相信，当年轻患者仍然拥有大量肌肉时，尽早治疗将会使患者获得最大益处”。

杜兴氏肌肉营养不良症（Duchenne Muscular Dystrophy，DMD）是一种相当严重的性联遗传肌肉失养症。男性病患大约在4岁开始就会产生肌肉无力的症状，此后症状即会开始快速恶化。通常最先从大腿即骨盆肌肉开始萎缩，之后则是上臂肌肉。本病会导致站立困难，患者大约在12岁之后就无法行走。


原始出处：

http://www.firstwordpharma.com/node/1646096#axzz5qOw0cfEI

本文系梅斯医学（MedSci）原创编译整理，转载需授权!

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 (3)
#插入话题
  1. [GetPortalCommentsPageByObjectIdResponse(id=1714834, encodeId=72a41e148348c, content=<a href='/topic/show?id=021230e312' target=_blank style='color:#2F92EE;'>#AZ#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=22, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=3073, encryptionId=021230e312, topicName=AZ)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=9e5031899089, createdName=12498e5fm22(暂无昵称), createdTime=Sat Nov 09 17:09:00 CST 2019, time=2019-11-09, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1839696, encodeId=e77b1839696e8, content=<a href='/topic/show?id=341ae311ea' target=_blank style='color:#2F92EE;'>#FDA批准#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=17, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=7311, encryptionId=341ae311ea, topicName=FDA批准)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=4dfa37, createdName=智者为医08, createdTime=Mon Oct 14 08:09:00 CST 2019, time=2019-10-14, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1545525, encodeId=ab4d154552586, content=<a href='/topic/show?id=e9d5604489' target=_blank style='color:#2F92EE;'>#DMD#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=26, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=6044, encryptionId=e9d5604489, topicName=DMD)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=57b213767844, createdName=紫砂壶, createdTime=Wed Jun 12 11:09:00 CST 2019, time=2019-06-12, status=1, ipAttribution=)]
    2019-11-09 12498e5fm22(暂无昵称)

    #AZ#

    0

  2. [GetPortalCommentsPageByObjectIdResponse(id=1714834, encodeId=72a41e148348c, content=<a href='/topic/show?id=021230e312' target=_blank style='color:#2F92EE;'>#AZ#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=22, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=3073, encryptionId=021230e312, topicName=AZ)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=9e5031899089, createdName=12498e5fm22(暂无昵称), createdTime=Sat Nov 09 17:09:00 CST 2019, time=2019-11-09, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1839696, encodeId=e77b1839696e8, content=<a href='/topic/show?id=341ae311ea' target=_blank style='color:#2F92EE;'>#FDA批准#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=17, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=7311, encryptionId=341ae311ea, topicName=FDA批准)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=4dfa37, createdName=智者为医08, createdTime=Mon Oct 14 08:09:00 CST 2019, time=2019-10-14, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1545525, encodeId=ab4d154552586, content=<a href='/topic/show?id=e9d5604489' target=_blank style='color:#2F92EE;'>#DMD#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=26, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=6044, encryptionId=e9d5604489, topicName=DMD)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=57b213767844, createdName=紫砂壶, createdTime=Wed Jun 12 11:09:00 CST 2019, time=2019-06-12, status=1, ipAttribution=)]
    2019-10-14 智者为医08

    #FDA批准#

    0

  3. [GetPortalCommentsPageByObjectIdResponse(id=1714834, encodeId=72a41e148348c, content=<a href='/topic/show?id=021230e312' target=_blank style='color:#2F92EE;'>#AZ#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=22, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=3073, encryptionId=021230e312, topicName=AZ)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=9e5031899089, createdName=12498e5fm22(暂无昵称), createdTime=Sat Nov 09 17:09:00 CST 2019, time=2019-11-09, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1839696, encodeId=e77b1839696e8, content=<a href='/topic/show?id=341ae311ea' target=_blank style='color:#2F92EE;'>#FDA批准#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=17, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=7311, encryptionId=341ae311ea, topicName=FDA批准)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=4dfa37, createdName=智者为医08, createdTime=Mon Oct 14 08:09:00 CST 2019, time=2019-10-14, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1545525, encodeId=ab4d154552586, content=<a href='/topic/show?id=e9d5604489' target=_blank style='color:#2F92EE;'>#DMD#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=26, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=6044, encryptionId=e9d5604489, topicName=DMD)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=57b213767844, createdName=紫砂壶, createdTime=Wed Jun 12 11:09:00 CST 2019, time=2019-06-12, status=1, ipAttribution=)]
    2019-06-12 紫砂壶

    #DMD#

    0