Gruskin, D.

patient study disease treatment pompe infantile-onset pompe disease group motor alglucosidase alfa survival rhgaa baseline ventilation pompe disease 18 patients death alglucosidase glycogen month muscle control group age 36 mo 18 months control infantile-onset extension study antibody skill assessment kishnani acid ␣- glucosidase alglucosidase alfa treatment van der ploeg table cardiomyopathy activity infusion function genzyme corporation ventilator use rhgaa treatment trial analysis figure event status therapy fi nal assessment level enzyme ventilator infant department extension motor skills 40 mg /kg response support motor function ventilatory support pediatrics glucosidase neurology week 52 content development university crim-negative patients symptom muscle glycogen content z-score age 24 mo patient c patients c glycogen content 6 months motor development children efficacy safety