The Global Perspective on Respiratory Care for Neuromuscular Disease
摘要
Standards of care protocols and rare disease networks have improved the quality of care and research internationally for people with neuromuscular disease (NMD) over the last 20–30 years. These developments have been associated with a global increase in survival and prevalence of people with NMD, largely due to comprehensive medical care and optimal ventilatory support. Advances in gene therapy, gene editing and other pharmacological interventions now promise to improve outcomes further, but the healthcare impacts of these require detailed assessment, and long-term outcomes remain unclear.