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美国批准PKU酶药物

2018-05-25 MedSci MedSci原创

PKU酶药物
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Biomarin的Palynziq已被批准为首个用于治疗美国成年人罕见的脑部威胁性遗传病苯丙酮尿症（PKU）的酶疗法。Palynziq（pegvaliase-pqpz）是一种聚乙二醇重组苯丙氨酸解氨酶，它是通过帮助机体恢复其分解Phe（一种在所有形式的蛋白质中发现的氨基酸）的能力来靶向PKU潜在原因的唯一治疗方法。高水平的苯丙氨酸会对大脑产生毒性，并可能导致严重的神经和精神问题，影响人们的思维，感


Biomarin的Palynziq已被批准为首个用于治疗美国成年人罕见的脑部威胁性遗传病苯丙酮尿症（PKU）的酶疗法。

Palynziq（pegvaliase-pqpz）是一种聚乙二醇重组苯丙氨酸解氨酶，它是通过帮助机体恢复其分解Phe（一种在所有形式的蛋白质中发现的氨基酸）的能力来靶向PKU潜在原因的唯一治疗方法。

高水平的苯丙氨酸会对大脑产生毒性，并可能导致严重的神经和精神问题，影响人们的思维，感觉和行为。

PKU患者需要终身管理，包括坚持严格限制日常饮食的医疗食品和配方，以避免摄入大多数食物中存在的Phe。

Palynziq的批准"标志着成年人生活的重要里程碑"，该公司现在可以获得有效的新治疗方案来控制他们的血液Phe，"该公司指出。

北京大学联盟执行主任Christine Brown表示："尽管经过严格的管理，Palynziq有可能成为北大成年人改变游戏规则的治疗方式，尽管他们一生都在努力控制其Phe水平。

该疗法目前也在欧洲进行审查。


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  1. [GetPortalCommentsPageByObjectIdResponse(id=2055042, encodeId=556620550423b, content=<a href='/topic/show?id=fa0ee98149c' target=_blank style='color:#2F92EE;'>#美国批准#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=62, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=79814, encryptionId=fa0ee98149c, topicName=美国批准)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=d47b2500175, createdName=12498cf9m35(暂无昵称), createdTime=Wed Aug 15 00:17:00 CST 2018, time=2018-08-15, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=318708, encodeId=2977318e08a3, content=学习谢谢分享, beContent=null, objectType=article, channel=null, level=null, likeNumber=68, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/mStl88fu4Nd44v3xHnnictn9UpctwtH62EPQ59TMdI9lnqHMgQ16nH7GmGCfpzicsHgjibYVk6vwSpXrC86gMibRkoaIzf5HwSz7/0, createdBy=78cc1691107, createdName=龙胆草, createdTime=Sat May 26 09:40:00 CST 2018, time=2018-05-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=318662, encodeId=05ec31866222, content=阅, beContent=null, objectType=article, channel=null, level=null, likeNumber=88, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/vi_32/fhZdOu27e88Voqh19n8eF8cLplo8L4nicKtH0WnE3KFELXns5XDu8rsiceK2HHDZc2SEStia5VV3Pia2QLMVqlWbHw/0, createdBy=6e012139475, createdName=神功盖世, createdTime=Sat May 26 07:40:03 CST 2018, time=2018-05-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=318632, encodeId=21113186320d, content=学习了.谢谢分享, beContent=null, objectType=article, channel=null, level=null, likeNumber=77, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=7c592194322, createdName=1209e435m98(暂无昵称), createdTime=Sat May 26 07:10:25 CST 2018, time=2018-05-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=318616, encodeId=12a731861629, content=厉害了我的哥, beContent=null, objectType=article, channel=null, level=null, likeNumber=70, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/oLAjfB7s1ib3IzoRn621JdKCFUI4XvNELcibocrAVU6aUzEVDSaf3rYH08PUYUiagGqxAEhj8g9WqqNvD1CupIpCrmnR2OrqHZh/0, createdBy=7f181365728, createdName=飛歌, createdTime=Sat May 26 06:51:41 CST 2018, time=2018-05-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=318602, encodeId=d0143186028a, content=学习学习学习学习, beContent=null, objectType=article, channel=null, level=null, likeNumber=86, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/mStl88fu4NfNLvzZhgPxRkia1DP5MpwEgtXo1XhXA11xD6MWKxunAZFXKSNic0fWHUQx83oNHPlRMxxIctM83QKeCLkJpMibibiaC/0, createdBy=7e581734408, createdName=深海的鱼, createdTime=Sat May 26 06:12:01 CST 2018, time=2018-05-26, status=1, ipAttribution=)]
    2018-08-15 12498cf9m35(暂无昵称)

    #美国批准#

    0

  2. [GetPortalCommentsPageByObjectIdResponse(id=2055042, encodeId=556620550423b, content=<a href='/topic/show?id=fa0ee98149c' target=_blank style='color:#2F92EE;'>#美国批准#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=62, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=79814, encryptionId=fa0ee98149c, topicName=美国批准)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=d47b2500175, createdName=12498cf9m35(暂无昵称), createdTime=Wed Aug 15 00:17:00 CST 2018, time=2018-08-15, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=318708, encodeId=2977318e08a3, content=学习谢谢分享, beContent=null, objectType=article, channel=null, level=null, likeNumber=68, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/mStl88fu4Nd44v3xHnnictn9UpctwtH62EPQ59TMdI9lnqHMgQ16nH7GmGCfpzicsHgjibYVk6vwSpXrC86gMibRkoaIzf5HwSz7/0, createdBy=78cc1691107, createdName=龙胆草, createdTime=Sat May 26 09:40:00 CST 2018, time=2018-05-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=318662, encodeId=05ec31866222, content=阅, beContent=null, objectType=article, channel=null, level=null, likeNumber=88, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/vi_32/fhZdOu27e88Voqh19n8eF8cLplo8L4nicKtH0WnE3KFELXns5XDu8rsiceK2HHDZc2SEStia5VV3Pia2QLMVqlWbHw/0, createdBy=6e012139475, createdName=神功盖世, createdTime=Sat May 26 07:40:03 CST 2018, time=2018-05-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=318632, encodeId=21113186320d, content=学习了.谢谢分享, beContent=null, objectType=article, channel=null, level=null, likeNumber=77, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=7c592194322, createdName=1209e435m98(暂无昵称), createdTime=Sat May 26 07:10:25 CST 2018, time=2018-05-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=318616, encodeId=12a731861629, content=厉害了我的哥, beContent=null, objectType=article, channel=null, level=null, likeNumber=70, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/oLAjfB7s1ib3IzoRn621JdKCFUI4XvNELcibocrAVU6aUzEVDSaf3rYH08PUYUiagGqxAEhj8g9WqqNvD1CupIpCrmnR2OrqHZh/0, createdBy=7f181365728, createdName=飛歌, createdTime=Sat May 26 06:51:41 CST 2018, time=2018-05-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=318602, encodeId=d0143186028a, content=学习学习学习学习, beContent=null, objectType=article, channel=null, level=null, likeNumber=86, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/mStl88fu4NfNLvzZhgPxRkia1DP5MpwEgtXo1XhXA11xD6MWKxunAZFXKSNic0fWHUQx83oNHPlRMxxIctM83QKeCLkJpMibibiaC/0, createdBy=7e581734408, createdName=深海的鱼, createdTime=Sat May 26 06:12:01 CST 2018, time=2018-05-26, status=1, ipAttribution=)]
    2018-05-26 龙胆草

    学习谢谢分享

    0

  3. [GetPortalCommentsPageByObjectIdResponse(id=2055042, encodeId=556620550423b, content=<a href='/topic/show?id=fa0ee98149c' target=_blank style='color:#2F92EE;'>#美国批准#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=62, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=79814, encryptionId=fa0ee98149c, topicName=美国批准)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=d47b2500175, createdName=12498cf9m35(暂无昵称), createdTime=Wed Aug 15 00:17:00 CST 2018, time=2018-08-15, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=318708, encodeId=2977318e08a3, content=学习谢谢分享, beContent=null, objectType=article, channel=null, level=null, likeNumber=68, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/mStl88fu4Nd44v3xHnnictn9UpctwtH62EPQ59TMdI9lnqHMgQ16nH7GmGCfpzicsHgjibYVk6vwSpXrC86gMibRkoaIzf5HwSz7/0, createdBy=78cc1691107, createdName=龙胆草, createdTime=Sat May 26 09:40:00 CST 2018, time=2018-05-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=318662, encodeId=05ec31866222, content=阅, beContent=null, objectType=article, channel=null, level=null, likeNumber=88, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/vi_32/fhZdOu27e88Voqh19n8eF8cLplo8L4nicKtH0WnE3KFELXns5XDu8rsiceK2HHDZc2SEStia5VV3Pia2QLMVqlWbHw/0, createdBy=6e012139475, createdName=神功盖世, createdTime=Sat May 26 07:40:03 CST 2018, time=2018-05-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=318632, encodeId=21113186320d, content=学习了.谢谢分享, beContent=null, objectType=article, channel=null, level=null, likeNumber=77, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=7c592194322, createdName=1209e435m98(暂无昵称), createdTime=Sat May 26 07:10:25 CST 2018, time=2018-05-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=318616, encodeId=12a731861629, content=厉害了我的哥, beContent=null, objectType=article, channel=null, level=null, likeNumber=70, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/oLAjfB7s1ib3IzoRn621JdKCFUI4XvNELcibocrAVU6aUzEVDSaf3rYH08PUYUiagGqxAEhj8g9WqqNvD1CupIpCrmnR2OrqHZh/0, createdBy=7f181365728, createdName=飛歌, createdTime=Sat May 26 06:51:41 CST 2018, time=2018-05-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=318602, encodeId=d0143186028a, content=学习学习学习学习, beContent=null, objectType=article, channel=null, level=null, likeNumber=86, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/mStl88fu4NfNLvzZhgPxRkia1DP5MpwEgtXo1XhXA11xD6MWKxunAZFXKSNic0fWHUQx83oNHPlRMxxIctM83QKeCLkJpMibibiaC/0, createdBy=7e581734408, createdName=深海的鱼, createdTime=Sat May 26 06:12:01 CST 2018, time=2018-05-26, status=1, ipAttribution=)]
    2018-05-26 神功盖世

    阅

    0

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    2018-05-26 1209e435m98(暂无昵称)

    学习了.谢谢分享

    0

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    2018-05-26 飛歌

    厉害了我的哥

    0

  6. [GetPortalCommentsPageByObjectIdResponse(id=2055042, encodeId=556620550423b, content=<a href='/topic/show?id=fa0ee98149c' target=_blank style='color:#2F92EE;'>#美国批准#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=62, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=79814, encryptionId=fa0ee98149c, topicName=美国批准)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=d47b2500175, createdName=12498cf9m35(暂无昵称), createdTime=Wed Aug 15 00:17:00 CST 2018, time=2018-08-15, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=318708, encodeId=2977318e08a3, content=学习谢谢分享, beContent=null, objectType=article, channel=null, level=null, likeNumber=68, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/mStl88fu4Nd44v3xHnnictn9UpctwtH62EPQ59TMdI9lnqHMgQ16nH7GmGCfpzicsHgjibYVk6vwSpXrC86gMibRkoaIzf5HwSz7/0, createdBy=78cc1691107, createdName=龙胆草, createdTime=Sat May 26 09:40:00 CST 2018, time=2018-05-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=318662, encodeId=05ec31866222, content=阅, beContent=null, objectType=article, channel=null, level=null, likeNumber=88, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/vi_32/fhZdOu27e88Voqh19n8eF8cLplo8L4nicKtH0WnE3KFELXns5XDu8rsiceK2HHDZc2SEStia5VV3Pia2QLMVqlWbHw/0, createdBy=6e012139475, createdName=神功盖世, createdTime=Sat May 26 07:40:03 CST 2018, time=2018-05-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=318632, encodeId=21113186320d, content=学习了.谢谢分享, beContent=null, objectType=article, channel=null, level=null, likeNumber=77, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=7c592194322, createdName=1209e435m98(暂无昵称), createdTime=Sat May 26 07:10:25 CST 2018, time=2018-05-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=318616, encodeId=12a731861629, content=厉害了我的哥, beContent=null, objectType=article, channel=null, level=null, likeNumber=70, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/oLAjfB7s1ib3IzoRn621JdKCFUI4XvNELcibocrAVU6aUzEVDSaf3rYH08PUYUiagGqxAEhj8g9WqqNvD1CupIpCrmnR2OrqHZh/0, createdBy=7f181365728, createdName=飛歌, createdTime=Sat May 26 06:51:41 CST 2018, time=2018-05-26, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=318602, encodeId=d0143186028a, content=学习学习学习学习, beContent=null, objectType=article, channel=null, level=null, likeNumber=86, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/mStl88fu4NfNLvzZhgPxRkia1DP5MpwEgtXo1XhXA11xD6MWKxunAZFXKSNic0fWHUQx83oNHPlRMxxIctM83QKeCLkJpMibibiaC/0, createdBy=7e581734408, createdName=深海的鱼, createdTime=Sat May 26 06:12:01 CST 2018, time=2018-05-26, status=1, ipAttribution=)]
    2018-05-26 深海的鱼

    学习学习学习学习

    0

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