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肾上腺脑白质营养不良的Lenti-D基因治疗的全新数据将于SSIEM 2018研讨会上公布

2018-08-05 MedSci MedSci原创

SSIEM肾上腺脑白质营养不良基因治疗
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bluebird生物近日宣布，其用于研究儿童肾上腺脑白质营养不良（CALD）患者的Lenti-D基因疗法的临床试验的最新数据将在2018年SSIEM研讨会上公布。正在进行的II/III期Starbeam研究（ALD-102）的更新数据将评估Lenti-D基因治疗在17岁及以下CALD患者中的有效性和安全性。该公司还将提供其正在进行的观察性研究（ALD-103）的同种异体造血干细胞移植（allo-H

bluebird生物近日宣布，其用于研究儿童肾上腺脑白质营养不良（CALD）患者的Lenti-D基因疗法的临床试验的最新数据将在2018年SSIEM研讨会上公布。正在进行的II/III期Starbeam研究（ALD-102）的更新数据将评估Lenti-D基因治疗在17岁及以下CALD患者中的有效性和安全性。该公司还将提供其正在进行的观察性研究（ALD-103）的同种异体造血干细胞移植（allo-HSCT）在17岁及以下CALD患者中的初步数据。

CALD是一种临床异质性很强的遗传性代谢性疾病。表现在同一家系可有不同表型，同一患者不同时期表现也不同。根据ALD的发病年龄及临床表现分为7型：儿童脑型、青少年脑型、成人脑型、肾上腺脊髓神经病型(Adrenomyeloneuropathy， AMN)、Addison 型、无症状型和杂合子型。

bluebird生物公司首席医疗官David Davidson博士说：“我们继续推进针对CALD的Lenti-D基因治疗的开发计划，并很高兴有机会在SSIEM分享我们的两项研究中的最新数据。除了更新ALD-102治疗研究的临床结果外，我们还将首次提供ALD-103观察性研究的中期数据，ALD-103研究是针对CALD进行的同种异体造血干细胞移植治疗”。


原始出处：

http://www.firstwordpharma.com/node/1583086?tsid=28®ion_id=4#axzz5NCMJrdAy

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    2018-12-15 nymo

    #SSI#

    0

  2. [GetPortalCommentsPageByObjectIdResponse(id=1649426, encodeId=cc781649426e7, content=<a href='/topic/show?id=081a16e10e8' target=_blank style='color:#2F92EE;'>#SSI#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=58, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=16710, encryptionId=081a16e10e8, topicName=SSI)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=586523824746, createdName=nymo, createdTime=Sat Dec 15 01:22:00 CST 2018, time=2018-12-15, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1895142, encodeId=ccb31895142d7, content=<a href='/topic/show?id=ce478241e0b' target=_blank style='color:#2F92EE;'>#肾上腺#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=48, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=82417, encryptionId=ce478241e0b, topicName=肾上腺)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=8dba135, createdName=xuyong523, createdTime=Sun Oct 07 05:22:00 CST 2018, time=2018-10-07, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1994808, encodeId=254619948082e, content=<a href='/topic/show?id=3d51e345936' target=_blank style='color:#2F92EE;'>#研讨会#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=50, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=73459, encryptionId=3d51e345936, topicName=研讨会)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=87b1109, createdName=kcb069, createdTime=Thu Jun 20 01:22:00 CST 2019, time=2019-06-20, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1765732, encodeId=91481e65732ae, content=<a href='/topic/show?id=252ae1803a3' target=_blank style='color:#2F92EE;'>#白质#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=54, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=71803, encryptionId=252ae1803a3, topicName=白质)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=778d36, createdName=柳叶一刀, createdTime=Wed Aug 22 19:22:00 CST 2018, time=2018-08-22, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=336641, encodeId=e76333664198, content=学习一下，很有意义, beContent=null, objectType=article, channel=null, level=null, likeNumber=84, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c33e2412978, createdName=1237db7em74暂无昵称, createdTime=Sun Aug 05 13:50:49 CST 2018, time=2018-08-05, status=1, ipAttribution=)]
    2018-10-07 xuyong523

    #肾上腺#

    0

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    2019-06-20 kcb069

    #研讨会#

    0

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    2018-08-22 柳叶一刀

    #白质#

    0

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    2018-08-05 1237db7em74暂无昵称

    学习一下，很有意义

    0