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Glycogen storage disease type II, also known as Pompe disease, is a rare, progressive disorder. It is characterized by partial or total deficiency of acid alpha-glucosidase (GAA), an enzyme that hydrolyzes lysosomal glycogen.1) As a result of deficiency of GAA enzyme, lysosomal glycogen accumulates in tissues of the body, which can lead to progressive debilitation, organ failure, and death. The diagnosis of Pompe disease in children and adults can be challenging because of the heterogeneous clinical presentation and considerable overlap of signs and symptoms identified in other neuromuscular diseases.1) The infantile-onset form of the disease is rapidly progressive which is characterized by marked cardiomyopathy, respiratory failure, and death within the first year of life.2) Late-onset Pompe disease can present from the age of 1 year to the sixth decade of life which is characterized by progressive proximal skeletal muscle weakness and respiratory insufficiency that leads to respiratory failure and death.3,4) Enzyme replacement therapy (ERT) using recombinant human acid alpha-glucosidase was developed as the most promising therapeutic approach for Pompe disease al though the clinical response is variable; however, the evidence is lacking, especially regarding the optimal dose or dose frequency and the effect of long-term alglucosidase alfa treatment on extended survival as well as ventilation-free survival and cardiomyopathy.5) Recently, Kim et al.6) published clinical features, genotypes, changes before and after ERT, and long-term outcomes in 5 Korean patients with infantile-onset Pompe disease (IOPD) and late-onset Pompe disease (LOPD). During a median 10-year ERT, there remains stable in the abnormalities shown on initial echocardiogram in patients with IOPD, along with improved organomegaly and normalization of liver transaminase level in patients with late-onset Pompe disease without adverse effects. Usually, clinical effectiveness in Pompe disease is evaluated by ventilator-free survival, reduction in left ventri cular mass index, and improvement of motor function. The study by Kim et al.6) was well conducted on this score to assess clinical effectiveness including quality of life in patients with Pompe disease. To evaluate the functional exercise capacity, there are several Considerations for evaluating the effectiveness and longterm outcome of enzyme replacement therapy in Pompe disease

Considerations for evaluating the effectiveness and long-term outcome of enzyme replacement therapy in Pompe disease