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黏多糖贮积症（Mucopolysaccharidsis，MPS）基因治疗计划取得积极中期数据

2020-02-13 Allan MedSci原创

黏多糖贮积症MPSABO-102ABO-101
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基因和细胞治疗领域的领导者Abeona Therapeutics近日宣布，两个正在进行的I / II期临床试验（Transpher A研究和B研究）的积极中期数据将在第16届WORLDSymposium™上公布。

基因和细胞治疗领域的领导者Abeona Therapeutics近日宣布，两个正在进行的I / II期临床试验（Transpher A研究和B研究）的积极中期数据将在第16届WORLDSymposium™上公布：1）Transpher A研究的结果表明，ABO-102能够改善年龄小于30个月的MPS IIIA患者的神经认知。治疗两年后，脑脊液（CSF）硫酸乙酰肝素（HS）水平和肝脏体积保持在稳定水平；2）Transpher B研究的结果表明，ABO-101改善了多种疾病生物标志物，为MPS IIIB患者的生物学效应提供了明确的证据。第2组的给药已完成，第3组的第一例患者在2020年1月下旬接受了治疗，迄今为止共有8例患者接受了治疗。

黏多糖贮积症（Muco polysaccharidsis，MPS）是由于人体细胞的溶酶体内降解黏多糖的水解酶发生突变导致其活性丧失，黏多糖不能被降解代谢，最终贮积在体内而发生的疾病。


原始出处：

https://www.firstwordpharma.com/node/1700298?tsid=28®ion_id=5

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    2020-12-02 lifefamily

    #黏多糖贮积症#好象不少见哦，是一种较多的#罕见病#

    0

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    2020-04-17 124987e8m07暂无昵称

    #MPS#

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    2020-08-10 12498e67m28暂无昵称

    #治疗计划#

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    2020-02-15 12498568m50暂无昵称

    #ACC#

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