Catalyst制药宣布开展Firdapse治疗MuSK抗体阳性重症肌无力的IIb期临床试验

2019-01-06 MedSci MedSci原创

Catalyst是一家生物制药公司,专注于为患有罕见的慢性神经疾病的患者开发和商业化创新疗法,Catalyst近日公布了Firdapse(磷酸氨苄西林)治疗MuSK抗体阳性重症肌无力(MuSK-MG)的IIb期临床试验(MSK-001)的最新结果。

Catalyst是一家生物制药公司,专注于为患有罕见的慢性神经疾病的患者开发和商业化创新疗法,Catalyst近日公布了Firdapse(磷酸氨苄西林)治疗MuSK抗体阳性重症肌无力(MuSK-MG)的IIb临床试验(MSK-001)的最新结果。

MSK-001试验是一项随机、双盲、安慰剂对照、双交叉设计的临床试验,结果显示,磷酸氨苄西林达到了主要终点(QMG评分p = 0.0003MG日常生活概况评分p = 0.0006)以及次要终点。该研究提供的证据表明磷酸氨苄西林在治疗MuSK-MG患者时是安全有效的。

重症肌无力是长期的神经肌肉疾病,会造成不同程度的肌肉无力。最常影响眼部、脸部及吞咽相关的肌肉。会造成复视、眼睑下垂、说话困难及行走困难等症状。重症肌无力可能会突然发生。患者多半有胸腺过大或是胸腺瘤的情形。如果重症肌无力 (MG) 患者血清肌肉特异性激酶 (MuSK) 抗体呈阳性则其症状可能更为严重,达到缓解的目标可能更难。


原始出处:

http://www.firstwordpharma.com/node/1615906?tsid=4#axzz5bnF206kJ

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    2019-11-29 bioon3
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    2019-01-16 jyzxjiangqin

    重症肌无力的治疗。

    0

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    2019-01-08 bbjsj_1981
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    2019-01-08 一叶知秋