神经肌肉疾病正受益于基因疗法、RNA疗法、免疫疗法、精准医疗和康复治疗的进步。本专题欢迎病例报告和基于病例的研究,描述新型疗法、意外疗效、不良事件和长期预后。主题包括肌营养不良症、脊髓性肌萎缩症(SMA)、肌萎缩侧索硬化症(ALS)、重症肌无力、周围神经病变、生物标志物、疾病机制和治疗决策。
Neuromuscular diseases are benefiting from advances in gene therapies, RNA-based treatments, immunotherapies, precision medicine, and rehabilitation. This Collection welcomes case reports and case-based studies describing novel therapies, unexpected responses, adverse events, and long-term outcomes. Topics include muscular dystrophies, SMA, ALS, myasthenia gravis, peripheral neuropathies, biomarkers, disease mechanisms, and treatment decision-making.