Corzo, D.

patient disease pompe treatment study muscle pompe disease group baseline month function rhgaa motor infantile-onset pompe disease 18 months glycogen therapy cohort van der ploeg enzyme activity control group 18 patients neurology control rhgaa treatment symptom trial survival strength table children center muscle strength death safety position infantile-onset erasmus mc university score supine position department assessment range university support a-glucosidase antibody genzyme corporation pediatrics enzyme replacement therapy enzyme therapy ventilatory support ventilation analysis development week 52 cohort 1 content ventilator use van capelle skill muscle glycogen content fi rst symptoms acid ␣- glucosidase value motor function test figure glycogen content 6 months supine level kishnani motor development infusion efficacy result van doorn pa right-censored effect