Smeitink, J.A.

patient disease pompe glucosidase glycogen month dutch storage pompe disease study mutation enzyme muscle literature treatment dutch patients glycogen storage disease therapy rotterdam 4 patients dutch patient group glucosidase activity glycogenosis type ii enzyme replacement therapy rhaglu activity heart pediatrics rabbit milk erasmu van der ploeg deficiency development infant group rabbit netherland motor effect department hypertrophy erasmus rotterdam reuser reuser aj 40 mg /kg glycogenosi genet course production acid maltase deficiency 20 mg /kg hum mol genet weight replacement value symptom alpha-glucosidase muscle function 4 years protein erasmus mc-sophia function 1 year 12 weeks enzyme therapy report death baseline november lysosomal survival information fabry disease patient 3 infusion hospital 2 patients 110- kda precursor children tissue