O.23A 10 year prospective study on the effects of enzyme replacement therapy in adult Pompe patients

Enzyme-replacement therapy (ERT) with alglucosidase alfa has become standard of care in the treatment of patients with Pompe disease and is known to benefit walking ability, muscle strength and respiratory function in studies up to 5 years' follow-up. The aim of this study was to determine the effects of 10 years' ERT, focusing on individual variability in treatment response.