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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

本文系梅斯医学（MedSci）原创编译整理，转载需授权！

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    2018-12-15 nymo

    #SSI#

    0

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    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