罗氏的risdiplam在1型脊髓性肌萎缩症婴儿中展现出治疗潜力

2020-06-03 MedSci原创 MedSci原创

FIREFISH临床研究结果显示,罗氏的risdiplam对有症状的1型脊髓性肌萎缩症(SMA)的1-7个月婴儿治疗12个月后,患者的运动能力显着改善。

FIREFISH临床研究第2部分的结果显示,罗氏的risdiplam对有症状的1型脊髓性肌萎缩症(SMA)的1-7个月婴儿治疗12个月后,患者的运动能力显着改善。Risdiplam是一种运动神经元存活基因 SMN2 的前体 mRNA 剪接修饰剂,可增加 SMN 蛋白水平。

该研究达到了其主要终点,即根据BSID-III总运动量表评估,到第12个月时,有29%的婴儿在没有支撑的情况下坐了5秒钟。这是在1型SMA的治疗历史中从未实现的目标。

根据HINE-2量表的评估,有43.9%的婴儿可以将头保持直立,31.7%的婴儿可以侧向滚动,4.9%的婴儿可以在支撑下站立。

此外,12个月后存活的婴儿中95%(36/38)保持吞咽能力,89%(34/38)能够口服喂养。而在该疾病的治疗历史上,所有年龄大于12个月的1型SMA婴儿均需要喂养支持。

MDUK牛津大学神经肌肉疾病中心的FIREFISH研究人员Laurent Servais教授说:"鉴于入组的中位年龄为5.3个月,这些结果特别令人鼓舞。保持吞咽能力特别重要,因为它可以帮助婴儿喂养,并提示risdiplam对延髓功能有重大影响。"

FIREFISH研究中risdiplam的安全性与其已知的安全性一致,最常见的不良事件是上呼吸道感染(46.3%),肺炎(39%),发热(39%),便秘(19.5%),鼻咽炎(12.2%),鼻炎(12.2%)和腹泻(9.8%)。最常见的严重不良事件是肺炎(31.7%),细支气管炎(4.9%),呼吸衰竭(4.9%)和肌张力低下(4.9%)。

原始出处:

http://www.pharmatimes.com/news/roches_risdiplam_shows_promise_in_type_1_sma_1339249

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    2021-02-04 lxg951
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    2020-12-02 lifefamily

    #risdiplam#这个药物已在国内上市了,可以买得到了,用于#脊髓性肌萎缩症##罗氏#公司研发的

    0

  3. [GetPortalCommentsPageByObjectIdResponse(id=2057677, encodeId=3935205e67743, content=<a href='/topic/show?id=c02b106e29a' target=_blank style='color:#2F92EE;'>#LAM#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=23, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=10672, encryptionId=c02b106e29a, topicName=LAM)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=04ce158, createdName=lxg951, createdTime=Thu Feb 04 20:56:36 CST 2021, time=2021-02-04, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=904294, encodeId=d06a904294dc, content=<a href='/topic/show?id=8c60155009b' target=_blank style='color:#2F92EE;'>#risdiplam#</a>这个药物已在国内上市了,可以买得到了,用于<a href='/topic/show?id=60a184608ae' target=_blank style='color:#2F92EE;'>#脊髓性肌萎缩症#</a>,<a href='/topic/show?id=4c2ce960918' target=_blank style='color:#2F92EE;'>#罗氏#</a>公司研发的, beContent=null, objectType=article, channel=null, level=null, likeNumber=122, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=15500, encryptionId=8c60155009b, topicName=risdiplam), TopicDto(id=84608, encryptionId=60a184608ae, topicName=脊髓性肌萎缩症), TopicDto(id=79609, encryptionId=4c2ce960918, topicName=罗氏)], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20211210/ede9c51184aa4f54ae6c1164cfb8eeac/967dbf3eff2943c7bf3ccfec3a776ab0.JPG, createdBy=a33789389, createdName=lifefamily, createdTime=Wed Dec 02 13:31:37 CST 2020, time=2020-12-02, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1723511, encodeId=1c7d1e235117f, content=<a href='/topic/show?id=3e238460e7e' target=_blank style='color:#2F92EE;'>#脊髓性肌萎缩#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=37, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=84607, encryptionId=3e238460e7e, topicName=脊髓性肌萎缩)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=901033462428, createdName=soongzhihua, createdTime=Sat Mar 13 19:56:36 CST 2021, time=2021-03-13, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1929094, encodeId=d5891929094ea, content=<a href='/topic/show?id=73c268540' target=_blank style='color:#2F92EE;'>#1型脊髓性肌萎缩症#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=30, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=685, encryptionId=73c268540, topicName=1型脊髓性肌萎缩症)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c6fc262, createdName=zhangmingxiang, createdTime=Tue Jan 19 03:56:36 CST 2021, time=2021-01-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1695927, encodeId=deaa169592e56, content=<a href='/topic/show?id=03b98809697' target=_blank style='color:#2F92EE;'>#萎缩#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=22, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=88096, encryptionId=03b98809697, topicName=萎缩)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=53fc29868494, createdName=chendoc252, createdTime=Sat Aug 08 14:56:36 CST 2020, time=2020-08-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1503135, encodeId=8c9c1503135a3, content=<a href='/topic/show?id=a71481058ca' target=_blank style='color:#2F92EE;'>#肌萎缩#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=20, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81058, encryptionId=a71481058ca, topicName=肌萎缩)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=4c37265, createdName=zhaojie88, createdTime=Sun May 03 00:56:36 CST 2020, time=2020-05-03, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1514468, encodeId=536915144680a, content=<a href='/topic/show?id=1ae010211143' target=_blank style='color:#2F92EE;'>#髓性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=24, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=102111, encryptionId=1ae010211143, topicName=髓性)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=ae8810654266, createdName=more is better!, createdTime=Sun May 03 00:56:36 CST 2020, time=2020-05-03, status=1, ipAttribution=)]
  4. [GetPortalCommentsPageByObjectIdResponse(id=2057677, encodeId=3935205e67743, content=<a href='/topic/show?id=c02b106e29a' target=_blank style='color:#2F92EE;'>#LAM#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=23, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=10672, encryptionId=c02b106e29a, topicName=LAM)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=04ce158, createdName=lxg951, createdTime=Thu Feb 04 20:56:36 CST 2021, time=2021-02-04, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=904294, encodeId=d06a904294dc, content=<a href='/topic/show?id=8c60155009b' target=_blank style='color:#2F92EE;'>#risdiplam#</a>这个药物已在国内上市了,可以买得到了,用于<a href='/topic/show?id=60a184608ae' target=_blank style='color:#2F92EE;'>#脊髓性肌萎缩症#</a>,<a href='/topic/show?id=4c2ce960918' target=_blank style='color:#2F92EE;'>#罗氏#</a>公司研发的, beContent=null, objectType=article, channel=null, level=null, likeNumber=122, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=15500, encryptionId=8c60155009b, topicName=risdiplam), TopicDto(id=84608, encryptionId=60a184608ae, topicName=脊髓性肌萎缩症), TopicDto(id=79609, encryptionId=4c2ce960918, topicName=罗氏)], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20211210/ede9c51184aa4f54ae6c1164cfb8eeac/967dbf3eff2943c7bf3ccfec3a776ab0.JPG, createdBy=a33789389, createdName=lifefamily, createdTime=Wed Dec 02 13:31:37 CST 2020, time=2020-12-02, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1723511, encodeId=1c7d1e235117f, content=<a href='/topic/show?id=3e238460e7e' target=_blank style='color:#2F92EE;'>#脊髓性肌萎缩#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=37, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=84607, encryptionId=3e238460e7e, topicName=脊髓性肌萎缩)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=901033462428, createdName=soongzhihua, createdTime=Sat Mar 13 19:56:36 CST 2021, time=2021-03-13, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1929094, encodeId=d5891929094ea, content=<a href='/topic/show?id=73c268540' target=_blank style='color:#2F92EE;'>#1型脊髓性肌萎缩症#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=30, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=685, encryptionId=73c268540, topicName=1型脊髓性肌萎缩症)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c6fc262, createdName=zhangmingxiang, createdTime=Tue Jan 19 03:56:36 CST 2021, time=2021-01-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1695927, encodeId=deaa169592e56, content=<a href='/topic/show?id=03b98809697' target=_blank style='color:#2F92EE;'>#萎缩#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=22, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=88096, encryptionId=03b98809697, topicName=萎缩)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=53fc29868494, createdName=chendoc252, createdTime=Sat Aug 08 14:56:36 CST 2020, time=2020-08-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1503135, encodeId=8c9c1503135a3, content=<a href='/topic/show?id=a71481058ca' target=_blank style='color:#2F92EE;'>#肌萎缩#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=20, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81058, encryptionId=a71481058ca, topicName=肌萎缩)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=4c37265, createdName=zhaojie88, createdTime=Sun May 03 00:56:36 CST 2020, time=2020-05-03, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1514468, encodeId=536915144680a, content=<a href='/topic/show?id=1ae010211143' target=_blank style='color:#2F92EE;'>#髓性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=24, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=102111, encryptionId=1ae010211143, topicName=髓性)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=ae8810654266, createdName=more is better!, createdTime=Sun May 03 00:56:36 CST 2020, time=2020-05-03, status=1, ipAttribution=)]
  5. [GetPortalCommentsPageByObjectIdResponse(id=2057677, encodeId=3935205e67743, content=<a href='/topic/show?id=c02b106e29a' target=_blank style='color:#2F92EE;'>#LAM#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=23, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=10672, encryptionId=c02b106e29a, topicName=LAM)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=04ce158, createdName=lxg951, createdTime=Thu Feb 04 20:56:36 CST 2021, time=2021-02-04, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=904294, encodeId=d06a904294dc, content=<a href='/topic/show?id=8c60155009b' target=_blank style='color:#2F92EE;'>#risdiplam#</a>这个药物已在国内上市了,可以买得到了,用于<a href='/topic/show?id=60a184608ae' target=_blank style='color:#2F92EE;'>#脊髓性肌萎缩症#</a>,<a href='/topic/show?id=4c2ce960918' target=_blank style='color:#2F92EE;'>#罗氏#</a>公司研发的, beContent=null, objectType=article, channel=null, level=null, likeNumber=122, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=15500, encryptionId=8c60155009b, topicName=risdiplam), TopicDto(id=84608, encryptionId=60a184608ae, topicName=脊髓性肌萎缩症), TopicDto(id=79609, encryptionId=4c2ce960918, topicName=罗氏)], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20211210/ede9c51184aa4f54ae6c1164cfb8eeac/967dbf3eff2943c7bf3ccfec3a776ab0.JPG, createdBy=a33789389, createdName=lifefamily, createdTime=Wed Dec 02 13:31:37 CST 2020, time=2020-12-02, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1723511, encodeId=1c7d1e235117f, content=<a href='/topic/show?id=3e238460e7e' target=_blank style='color:#2F92EE;'>#脊髓性肌萎缩#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=37, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=84607, encryptionId=3e238460e7e, topicName=脊髓性肌萎缩)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=901033462428, createdName=soongzhihua, createdTime=Sat Mar 13 19:56:36 CST 2021, time=2021-03-13, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1929094, encodeId=d5891929094ea, content=<a href='/topic/show?id=73c268540' target=_blank style='color:#2F92EE;'>#1型脊髓性肌萎缩症#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=30, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=685, encryptionId=73c268540, topicName=1型脊髓性肌萎缩症)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c6fc262, createdName=zhangmingxiang, createdTime=Tue Jan 19 03:56:36 CST 2021, time=2021-01-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1695927, encodeId=deaa169592e56, content=<a href='/topic/show?id=03b98809697' target=_blank style='color:#2F92EE;'>#萎缩#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=22, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=88096, encryptionId=03b98809697, topicName=萎缩)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=53fc29868494, createdName=chendoc252, createdTime=Sat Aug 08 14:56:36 CST 2020, time=2020-08-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1503135, encodeId=8c9c1503135a3, content=<a href='/topic/show?id=a71481058ca' target=_blank style='color:#2F92EE;'>#肌萎缩#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=20, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81058, encryptionId=a71481058ca, topicName=肌萎缩)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=4c37265, createdName=zhaojie88, createdTime=Sun May 03 00:56:36 CST 2020, time=2020-05-03, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1514468, encodeId=536915144680a, content=<a href='/topic/show?id=1ae010211143' target=_blank style='color:#2F92EE;'>#髓性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=24, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=102111, encryptionId=1ae010211143, topicName=髓性)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=ae8810654266, createdName=more is better!, createdTime=Sun May 03 00:56:36 CST 2020, time=2020-05-03, status=1, ipAttribution=)]
    2020-08-08 chendoc252
  6. [GetPortalCommentsPageByObjectIdResponse(id=2057677, encodeId=3935205e67743, content=<a href='/topic/show?id=c02b106e29a' target=_blank style='color:#2F92EE;'>#LAM#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=23, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=10672, encryptionId=c02b106e29a, topicName=LAM)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=04ce158, createdName=lxg951, createdTime=Thu Feb 04 20:56:36 CST 2021, time=2021-02-04, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=904294, encodeId=d06a904294dc, content=<a href='/topic/show?id=8c60155009b' target=_blank style='color:#2F92EE;'>#risdiplam#</a>这个药物已在国内上市了,可以买得到了,用于<a href='/topic/show?id=60a184608ae' target=_blank style='color:#2F92EE;'>#脊髓性肌萎缩症#</a>,<a href='/topic/show?id=4c2ce960918' target=_blank style='color:#2F92EE;'>#罗氏#</a>公司研发的, beContent=null, objectType=article, channel=null, level=null, likeNumber=122, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=15500, encryptionId=8c60155009b, topicName=risdiplam), TopicDto(id=84608, encryptionId=60a184608ae, topicName=脊髓性肌萎缩症), TopicDto(id=79609, encryptionId=4c2ce960918, topicName=罗氏)], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20211210/ede9c51184aa4f54ae6c1164cfb8eeac/967dbf3eff2943c7bf3ccfec3a776ab0.JPG, createdBy=a33789389, createdName=lifefamily, createdTime=Wed Dec 02 13:31:37 CST 2020, time=2020-12-02, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1723511, encodeId=1c7d1e235117f, content=<a href='/topic/show?id=3e238460e7e' target=_blank style='color:#2F92EE;'>#脊髓性肌萎缩#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=37, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=84607, encryptionId=3e238460e7e, topicName=脊髓性肌萎缩)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=901033462428, createdName=soongzhihua, createdTime=Sat Mar 13 19:56:36 CST 2021, time=2021-03-13, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1929094, encodeId=d5891929094ea, content=<a href='/topic/show?id=73c268540' target=_blank style='color:#2F92EE;'>#1型脊髓性肌萎缩症#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=30, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=685, encryptionId=73c268540, topicName=1型脊髓性肌萎缩症)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c6fc262, createdName=zhangmingxiang, createdTime=Tue Jan 19 03:56:36 CST 2021, time=2021-01-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1695927, encodeId=deaa169592e56, content=<a href='/topic/show?id=03b98809697' target=_blank style='color:#2F92EE;'>#萎缩#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=22, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=88096, encryptionId=03b98809697, topicName=萎缩)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=53fc29868494, createdName=chendoc252, createdTime=Sat Aug 08 14:56:36 CST 2020, time=2020-08-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1503135, encodeId=8c9c1503135a3, content=<a href='/topic/show?id=a71481058ca' target=_blank style='color:#2F92EE;'>#肌萎缩#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=20, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81058, encryptionId=a71481058ca, topicName=肌萎缩)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=4c37265, createdName=zhaojie88, createdTime=Sun May 03 00:56:36 CST 2020, time=2020-05-03, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1514468, encodeId=536915144680a, content=<a href='/topic/show?id=1ae010211143' target=_blank style='color:#2F92EE;'>#髓性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=24, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=102111, encryptionId=1ae010211143, topicName=髓性)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=ae8810654266, createdName=more is better!, createdTime=Sun May 03 00:56:36 CST 2020, time=2020-05-03, status=1, ipAttribution=)]
  7. [GetPortalCommentsPageByObjectIdResponse(id=2057677, encodeId=3935205e67743, content=<a href='/topic/show?id=c02b106e29a' target=_blank style='color:#2F92EE;'>#LAM#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=23, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=10672, encryptionId=c02b106e29a, topicName=LAM)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=04ce158, createdName=lxg951, createdTime=Thu Feb 04 20:56:36 CST 2021, time=2021-02-04, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=904294, encodeId=d06a904294dc, content=<a href='/topic/show?id=8c60155009b' target=_blank style='color:#2F92EE;'>#risdiplam#</a>这个药物已在国内上市了,可以买得到了,用于<a href='/topic/show?id=60a184608ae' target=_blank style='color:#2F92EE;'>#脊髓性肌萎缩症#</a>,<a href='/topic/show?id=4c2ce960918' target=_blank style='color:#2F92EE;'>#罗氏#</a>公司研发的, beContent=null, objectType=article, channel=null, level=null, likeNumber=122, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=15500, encryptionId=8c60155009b, topicName=risdiplam), TopicDto(id=84608, encryptionId=60a184608ae, topicName=脊髓性肌萎缩症), TopicDto(id=79609, encryptionId=4c2ce960918, topicName=罗氏)], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20211210/ede9c51184aa4f54ae6c1164cfb8eeac/967dbf3eff2943c7bf3ccfec3a776ab0.JPG, createdBy=a33789389, createdName=lifefamily, createdTime=Wed Dec 02 13:31:37 CST 2020, time=2020-12-02, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1723511, encodeId=1c7d1e235117f, content=<a href='/topic/show?id=3e238460e7e' target=_blank style='color:#2F92EE;'>#脊髓性肌萎缩#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=37, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=84607, encryptionId=3e238460e7e, topicName=脊髓性肌萎缩)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=901033462428, createdName=soongzhihua, createdTime=Sat Mar 13 19:56:36 CST 2021, time=2021-03-13, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1929094, encodeId=d5891929094ea, content=<a href='/topic/show?id=73c268540' target=_blank style='color:#2F92EE;'>#1型脊髓性肌萎缩症#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=30, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=685, encryptionId=73c268540, topicName=1型脊髓性肌萎缩症)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c6fc262, createdName=zhangmingxiang, createdTime=Tue Jan 19 03:56:36 CST 2021, time=2021-01-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1695927, encodeId=deaa169592e56, content=<a href='/topic/show?id=03b98809697' target=_blank style='color:#2F92EE;'>#萎缩#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=22, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=88096, encryptionId=03b98809697, topicName=萎缩)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=53fc29868494, createdName=chendoc252, createdTime=Sat Aug 08 14:56:36 CST 2020, time=2020-08-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1503135, encodeId=8c9c1503135a3, content=<a href='/topic/show?id=a71481058ca' target=_blank style='color:#2F92EE;'>#肌萎缩#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=20, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81058, encryptionId=a71481058ca, topicName=肌萎缩)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=4c37265, createdName=zhaojie88, createdTime=Sun May 03 00:56:36 CST 2020, time=2020-05-03, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1514468, encodeId=536915144680a, content=<a href='/topic/show?id=1ae010211143' target=_blank style='color:#2F92EE;'>#髓性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=24, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=102111, encryptionId=1ae010211143, topicName=髓性)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=ae8810654266, createdName=more is better!, createdTime=Sun May 03 00:56:36 CST 2020, time=2020-05-03, status=1, ipAttribution=)]

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脊髓性肌萎缩症(SMA):SMN2剪接修饰剂risdiplam显示出明显作用

罗氏制药公司近日公布了SUNFISH研究的第2部分的详细结果,该试验旨在评估实验性SMN2剪接修饰剂risdiplam治疗成人2型或3型脊髓性肌萎缩症(SMA)的有效性和安全性,结果表明,通过risdiplam的治疗,患者在运动功能的主要终点指标上均较基线有显著提高。