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面肩肱型肌营养不良症（FSHD）：Losmapimod的I期临床试验取得积极结果

2019-10-05 Allan MedSci原创

Losmapimod面肩肱型肌营养不良症FSHDa
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面肩肱型肌营养不良症（FSHD）是一种遗传性肌肉疾病，受其影响最严重的是脸、肩、上臂等部位的肌肉，该疾病是由骨骼肌细胞中DUX4的错误表达引起的。

面肩肱型肌营养不良症（FSHD）是一种遗传性肌肉疾病，受其影响最严重的是脸、肩、上臂等部位的肌肉，该疾病是由骨骼肌细胞中DUX4的错误表达引起的。专注于罕见疾病治疗的临床阶段生物制药公司Fulcrum近日宣布，选择性p38α/β丝裂原活化蛋白激酶（MAPK）抑制剂Losmapimod能够降低FSHD患者肌肉细胞中DUX4基因的表达。这项I期临床试验结果由Fulcrum Therapeutics的医学总监Michelle Mellion医师在丹麦哥本哈根举行的第24届世界肌肉协会国际年会上进行了口头报告。

Michelle Mellion医师表示：“Losmapimod在FSHD患者的I期临床试验中得出的初步结果表明，Losmapimod具有良好的耐受性，并在血浆和肌肉中达到了剂量依赖性浓度，根据临床前药理学研究认为该药物足以达到预期疗效”。


原始出处：

http://www.firstwordpharma.com/node/1671733#axzz61Rlned4y

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    2020-07-04 医生2402

    #I期临床#

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    2020-02-10 ms4468361851264128

    #肌营养不良症#

    0

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    2019-10-07 江川靖瑶

    #SMA#

    0

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    2019-10-07 july_981

    #Losmapimod#

    0

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    2019-10-05 坚强007

    向科研人员致敬！！！

    0

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