Ploeg, A.T. van der

patient disease pompe pompe disease muscle study enzyme therapy enzyme replacement therapy treatment glucosidase activity glycogen storage van der ploeg function lysosomal group fatigue children adult replacement month score disorder mps ii erasmus mc university netherland diagnosis result center reuser baseline erasmu syndrome level muscle strength range tissue scoliosi effect table ploeg university blood symptom mutation value infantile-onset pompe disease acid α- glucosidase hearing rotterdam motor alglucosidase alfa number analysis erasmus mc ficiency heart infusion van doorn pa department signi strength late-onset pompe disease mouse mucopolysaccharidosi genetic infant support reuser aj control genet protein weakness enzyme therapy change ventilation assessment alglucosidase