Abstract：
Duchenne muscular dystrophy (DMD) is a fatal genetic neuromuscular disorder, characterised by progressive decline in skeletal muscle function due to the secondary consequences of dystrophin deficiency. Weakness extends to the respiratory musculature, and cardiorespiratory failure is the leading cause of death in men with DMD. Intermittent hypoxia has emerged as a potential therapy to counteract ventilatory insufficiency by eliciting long-term facilitation of breathing. Mechanisms of sensory and motor facilitation of breathing have been well delineated in animal models. Various paradigms of intermittent hypoxia have been designed and implemented in human trials culminating in clinical trials in people with spinal cord injury and amyotrophic lateral sclerosis. Application of therapeutic intermittent hypoxia to DMD is considered together with discussion of the potential barriers to progression owing to the complexity of this devastating disease. Notwithstanding the considerable challenges and potential pitfalls of intermittent hypoxia-based therapies for DMD, we suggest it is incumbent on the research community to explore the potential benefits in pre-clinical models. Intermittent hypoxia paradigms should be implemented to explore the proclivity to express respiratory plasticity with the longer-term aim of preserving and potentiating ventilation in pre-clinical models and people with DMD.
摘要：
杜氏肌营养不良症（DMD）是一种致命的遗传性神经肌肉疾病，其特征是由于肌营养不良蛋白缺乏的继发性后果而导致骨骼肌功能逐渐下降。虚弱延伸到呼吸肌肉组织，和心肺功能衰竭是男性DMD的主要死亡原因。间歇性缺氧已成为一种潜在的疗法，可通过引起长期呼吸促进来抵消通气功能不全。在动物模型中已经很好地描述了感觉和运动促进呼吸的机制。已经在人体试验中设计并实施了间歇性缺氧的各种范例，最终在脊髓损伤和肌萎缩性侧索硬化症患者中进行了临床试验。考虑了治疗性间歇性缺氧对DMD的应用，并讨论了由于这种破坏性疾病的复杂性而导致的潜在进展障碍。尽管间歇性低氧治疗DMD存在相当大的挑战和潜在的缺陷，我们建议研究界有责任探索临床前模型的潜在益处.应实施间歇性缺氧范例，以探索表达呼吸可塑性的倾向，并以维持和增强临床前模型和DMD患者的通气为长期目标。
