McDonald, M.

patient disease treatment pompe rhgaa group study baseline month 18 months glycogen muscle motor infantile-onset pompe disease control group pompe disease 18 patients control rhgaa treatment survival death infantile-onset activity antibody trial genzyme corporation ventilatory support ventilation analysis neurology development week 52 content ventilator use skill muscle glycogen content acid ␣- glucosidase symptom van der ploeg figure glycogen content 6 months level kishnani motor development infusion efficacy safety 52 weeks criteria cardiomyopathy right-censored glycogen levels 20- tissue support table genzyme period article pediatrics glucosidase therapy 40- ventilator pompe pedi center response department function hospital ventilatory university enzyme corporation information failure assessment 40 mg /kg event