Beek, N.A.M.E. van der

patient disease pompe disease pompe muscle study function adult therapy muscle strength group treatment enzyme position children alglucosidase alfa strength van der ploeg glycogen range hearing storage activity supine position alglucosidase score scale van doorn pa course effect follow-up weakness late-onset pompe disease muscle weakness van der beek supine value sumscore result hearing loss motor duration glucosidase late-onset motor function test reuser enzyme-replacement therapy change diagnosis enzyme replacement therapy support reuser aj ploeg lysosomal measurement disorder table enzyme therapy disease duration deficiency mutation ventilation month metab erasmus mc university severity acid α- glucosidase difference disease progression mrc sumscore infantile-onset pompe disease wheelchair outcome decline symptom analysis involvement baseline hageman neurology