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一个试验性试验,以证实在GNE肌病中补充6'-sialyllactose的疗效:随机的,安慰剂对照试验

Young-Eun Park1, Jaeil Choi2, Lila Kim2

  • 1Department of Neurology, Pusan National University School of Medicine, Busan, Republic of Korea; Department of Neurology and Biomedical Research Institute, Pusan National University Hospital, Busan, Republic of Korea.

Molecular genetics and metabolism
|December 7, 2024
PubMed
概括

在GNE肌病症中补充6 - 黄乳糖 (6SL) 显示出减缓肌肉退化的潜力,这可以通过MRI脂肪分数的变化来证明. 虽然肌肉强度没有显著差异,6SL可能为这种罕见的遗传疾病提供长期益处.

关键词:
6′-sialyllactose 的使用情况.临床试验临床试验是指临床试验的临床试验.在GNE神经病变上.肌肉MRI是指肌肉的MRI.酸是酸的一种.

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