Department of Neurology, Yale School of Medicine, PO Box: 208018, New Haven, CT 06510, USA.
Center for Health + Technology, University of Rochester Medical Center, Rochester, NY 14642, USA.
Neurol Clin. 2021 Feb;39(1):87-112. doi: 10.1016/j.ncl.2020.09.005.
Increased understanding of disease pathophysiology and advances in gene therapies and drug technologies are revolutionizing treatment of muscular dystrophies and motor neuron disorders (MNDs). New drugs have been approved for Duchenne muscular dystrophy, spinal muscular atrophy, and amyotrophic lateral sclerosis. For other diseases, new targets have been identified, and new therapies are in clinical trials. The impact of such therapies will be fully understood only in the next decades. Cost burden and accessibility are major challenges in the wide application of new drugs. This article reviews advances in gene therapies, newly approved drugs, and therapeutic promises in muscular dystrophies and MNDs.
对疾病病理生理学的认识不断加深，以及基因疗法和药物技术的进步正在彻底改变肌肉萎缩症和运动神经元疾病（MND）的治疗方法。新药物已获准用于治疗杜氏肌营养不良症、脊髓性肌萎缩症和肌萎缩性侧索硬化症。对于其他疾病，已经确定了新的靶点，并且新的疗法正在临床试验中。这些疗法的影响只有在未来几十年才能完全理解。成本负担和可及性是新药物广泛应用的主要挑战。本文综述了肌肉萎缩症和 MND 中基因疗法、新批准药物和治疗前景的进展。
