Innovative RNA modifying and gene replacement therapies are currently revolutionizing the therapeutic landscape in 5q-associated spinal muscular atrophy (SMA). In order to provide individual recommendations for choice of treatment and therapy (dis-) continuation, objective outcome measures are needed. The purpose of this study was to determine whether maximum isometric voluntary grip and finger flexion strength is a useful sensitive outcome measure in children and adult patients with SMA. In this non-interventional, longitudinal pilot study, we assessed grip and finger flexion strength on 39 patients with SMA II and III (n = 16 children, mean age = 10.0; n = 23 adults, mean age = 38.4) using the Weber hand and finger dynamometer HFD 200. Grip and finger flexion strength, clinical examinations and motor function scores (Revised Upper Limb Module, Hammersmith Functional Motor Scale Expanded) were assessed over a 12-month treatment period concurrent with the nusinersen treatment. Grip and finger flexion strength was highly associated with motor function and disease severity, SMA type and copy number. During nusinersen treatment, grip and finger flexion strength significantly increased in children and adults with SMA. Grip and finger flexion strength measured with the HFD 200 is a promising sensitive outcome measure for SMA.

中文翻译：

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“读手掌”——握力和手指弯曲强度作为 5q 脊髓性肌萎缩症结果测量的初步研究

创新的 RNA 修饰和基因替代疗法目前正在彻底改变 5q 相关脊髓性肌萎缩症 (SMA) 的治疗格局。为了为治疗选择和治疗（终止）继续提供个体建议，需要客观的结果测量。本研究的目的是确定最大等长自愿握力和手指弯曲强度是否是 SMA 儿童和成人患者的有用的敏感结果测量。在这项非介入性纵向试点研究中，我们使用韦伯手评估了 39 名 SMA II 和 III 患者（n = 16 名儿童，平均年龄 = 10.0；n = 23 名成人，平均年龄 = 38.4）的握力和手指弯曲强度。和手指测力计 HFD 200。在 nusinersen 治疗的同时进行 12 个月的治疗期间，对握力和手指屈曲强度、临床检查和运动功能评分（修订版上肢模块、扩展哈默史​​密斯功能运动量表）进行了评估。握力和手指弯曲强度与运动功能和疾病严重程度、SMA 类型和拷贝数高度相关。在 nusinersen 治疗期间，患有 SMA 的儿童和成人的握力和手指弯曲强度显着增加。使用 HFD 200 测量的握力和手指弯曲强度是 SMA 的一项有前途的敏感结果测量方法。