Campanari Maria-Letizia, Bourefis Annis-Rayan, Kabashi Edor
Sorbonne Université, Université Pierre et Marie Curie, Université de Paris 06, Unité Mixte 75, Institut National de la Santé et de la Recherche Médicale (INSERM) Unité 1127, Centre National de la Recherche Scientifique, Unité Mixte de Recherche 7225 Institut du Cerveau et de la Moelle Épinière, Paris, France.
Imagine Institute, INSERM Unité 1163, Paris Descartes Université, Paris, France.
Front Neurol. 2019 Feb 6;10:68. doi: 10.3389/fneur.2019.00068. eCollection 2019.
Amyotrophic lateral sclerosis (ALS) represents the major adult-onset motor neuron disease. Both human and animal studies reveal the critical implication of muscle and neuromuscular junctions (NMJs) in the initial phase of this disease. Despite the common efforts, ALS diagnosis remains particularly challenging since many other disorders can overlap yielding similar clinical phenotypic features. A combination of further research on the NMJ parameters that are specific for this disease and laboratory tests are crucial for the early determination of specific changes in the muscle, as well as in motor neuron and the prediction of ALS progression. Also, it could provide a powerful tool in the discrimination of particular ALS and ALS-mimic cases and increase the efficacy of therapeutic treatments.
肌萎缩侧索硬化症(ALS)是主要的成人起病运动神经元疾病。人体和动物研究均表明,肌肉和神经肌肉接头(NMJ)在该疾病的初始阶段起着关键作用。尽管各方共同努力,但ALS的诊断仍然极具挑战性,因为许多其他疾病可能会出现重叠,产生相似的临床表型特征。进一步研究该疾病特有的NMJ参数并结合实验室检查,对于早期确定肌肉、运动神经元的特定变化以及预测ALS的进展至关重要。此外,它还可以为鉴别特定的ALS病例和疑似ALS病例提供有力工具,并提高治疗效果。