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Updated: Jul 13, 2026

Behavioral and Locomotor Measurements Using an Open Field Activity Monitoring System for Skeletal Muscle Diseases
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瑞卡尔S48168 (ARM210) 治疗与RYR1相关的神经病变:一期,开放标签,剂量升级试验

Joshua J Todd1,2,3, Tokunbor A Lawal2, Irene C Chrismer2

  • 1Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD 20814, USA.

EClinicalMedicine
|February 6, 2024
PubMed
概括

瑞卡尔S48168 (ARM210) 在患有RYR1相关肌肉病的患者中显示出良好的安全性和耐受性. 200毫克的剂量改善了疲劳和肌肉力量,为更大的试验铺平了道路.

关键词:
中心核心疾病 中心核心疾病生产性肌肉病变是一种先天性肌肉病变.

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