摘要
Duchenne型肌营养不良(DMD)是一种常见的X连锁隐性遗传致死性神经肌肉病.近年来基因相关治疗药物相继进入临床,全病程照护管理对提高患者生活质量和延长患者生命至关重要.为此,本共识通过系统查阅文献、专家论证、结合临床,最终形成对DMD不能独走期患者呼吸、心脏、康复、骨骼、营养、消化、皮肤、认知及心理等8个方面家庭照护的专家意见和建议,为DMD不能独走期患者的家庭照护提供指导与参考.
Abstract
Duchenne muscular dystrophy(DMD)is a severe X-linked recessive neuromuscular disorder characterized by progressive muscle weakness and eventual fatality.In recent years,more and more gene therapies have emerged,and comprehensive care throughout the disease course remains crucial for maximizing patient's survival and quality of life.This consensus,developed through literature review,expert consultations,and clinical experience,provides guidance for managing following aspects of care in non-ambulatory DMD patients,including respiratory,cardiac,rehabilitation,skeletal,nutritional,digestive,dermatological,cognitive,and psychological care.This aims to provide a scientific and practical support for families caring for non-ambulatory DMD patients.
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