Immune modulation in Pompe disease treated with enzyme replacement therapy

, many challenges remain. The development of an immune response with resultant loss of therapeu-tic efficacy of ERT remains a significant issue in Pompe disease, most notably in the infantile form. Similar obstacles have been faced in other conditions treated with therapeutic proteins, including hemo-philia, diabetes and multiple sclerosis, as well as lysosomal storage diseases like Fabry disease, Gaucher disease and the mucopolysaccharidoses