Most childhood neuromuscular disorders are caused by mutations causing abnormal expression or regulation of single genes or genetic pathways. The potential for gene therapy, gene editing and genetic therapies to ameliorate the course of these conditions is extraordinarily exciting, but there are significant challenges associated with their use, particularly with respect to safety, efficacy, cost and equity. Engagement with these novel technologies mandates careful assessment of the benefits and burdens of treatment for the patient, their family and their society. The examples provided by spinal muscular atrophy and Duchenne muscular dystrophy illustrate the potential value and challenges of gene and genetic therapies for paediatric neurological conditions. The cost and complexity of administration of these agents is a challenge for all countries. Jurisdictional variations in availability of newborn screening, genetic diagnostics, drug approval and reimbursement pathways, treatment and rehabilitation will affect equity of access, nationally and internationally. These challenges will best be addressed by collaboration by governments, pharma, clinicians and patient groups to establish frameworks for safe and cost-effective use of these exciting new therapies. No data are available. This is an invited review with no original data.

中文翻译：

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神经肌肉疾病的基因治疗：前景和伦理

大多数儿童神经肌肉疾病是由导致单个基因或遗传途径异常表达或调节的突变引起的。基因治疗、基因编辑和基因治疗改善这些疾病病程的潜力非常令人兴奋，但它们的使用存在重大挑战，特别是在安全性、有效性、成本和公平性方面。使用这些新技术要求仔细评估治疗对患者、他们的家人和他们的社会的益处和负担。脊髓性肌萎缩症和杜氏肌营养不良症提供的例子说明了基因和基因疗法对儿科神经系统疾病的潜在价值和挑战。这些药物的管理成本和复杂性对所有国家都是一个挑战。新生儿筛查、基因诊断、药物批准和报销途径、治疗和康复的可及性的管辖差异将影响国内和国际的公平性。这些挑战最好通过政府、制药公司、临床医生和患者团体的合作来解决，以建立安全且具有成本效益地使用这些令人兴奋的新疗法的框架。没有可用的数据。这是一个没有原始数据的邀请评论。临床医生和患者团体建立框架，以安全且经济高效地使用这些令人兴奋的新疗法。没有可用的数据。这是一个没有原始数据的邀请评论。临床医生和患者团体建立框架，以安全且经济高效地使用这些令人兴奋的新疗法。没有可用的数据。这是一个没有原始数据的邀请评论。