Hopkin, R.

patient disease baseline study treatment pompe alglucosidase alfa group alglucosidase rhgaa extension muscle month lots baseline pompe disease van der ploeg 18 months glycogen motor infantile-onset pompe disease late-onset pompe disease lots extension control group 18 patients antibody control rhgaa treatment safety university alglucosidase alfa treatment change neurology survival center late-onset therapy death infantile-onset activity table period hospital trial genzyme corporation ficacy result ventilatory support ventilation week 78 analysis function assessment percentage development department week 52 content ventilator use enzyme skill muscle glycogen content acid ␣- glucosidase symptom late-onset treatment study support placebo score 26 weeks figure glycogen content 6 months level kishnani motor development 6 mwt distance infusion efficacy genetic 52 weeks