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We investigated whether higher and more frequent dosing of alglucosidase alfa improves outcome.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Eight cross\u2010reactive immunological material (CRIM) positive patients were included in the study. All had fully deleterious mutations in both <jats:italic>GAA<\/jats:italic> alleles. Four received a dose of 20 mg\/kg every other week (eow) and four received 40 mg\/kg\/week. Survival, ventilator\u2010free survival, left\u2010ventricular mass index (LVMI), motor outcome, infusion\u2010associated reactions (IARs), and antibody formation were evaluated.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>All eight patients were alive at study end, seven of them remained ventilator\u2010free. The patient who became ventilator dependent was treated with 20 mg\/kg eow. Three of the four patients receiving 20 mg\/kg eow learned to walk; two of them maintained this ability at study end. All four patients receiving 40 mg\/kg\/week acquired and maintained the ability to walk at study end (ages of 3.3\u20135.6 years), even though their baseline motor functioning was poorer. There were no apparent differences between the two dose groups with respect to the effect of ERT on LVMI, the number of IARs and antibody formation.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Our data may suggest that a dose of 40 mg\/kg\/week improves outcome of CRIM positive patients over that brought by the currently recommended dose of 20 mg\/kg eow. Larger studies are needed to draw definite conclusions.<\/jats:p><\/jats:sec>","DOI":"10.1007\/s10545-015-9912-y","type":"journal-article","created":{"date-parts":[[2016,1,14]],"date-time":"2016-01-14T19:12:09Z","timestamp":1452798729000},"page":"383-390","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":69,"title":["Effects of a higher dose of alglucosidase alfa on ventilator\u2010free survival and motor outcome in classic infantile Pompe disease: an open\u2010label single\u2010center study"],"prefix":"10.1002","volume":"39","author":[{"given":"C. M.","family":"van Gelder","sequence":"first","affiliation":[{"name":"Department of Pediatrics, Division of Metabolic Diseases and Genetics, Center for Lysosomal and Metabolic Diseases Erasmus MC University Medical Center Dr Molewaterplein 60 Rotterdam 3015 GJ The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"E.","family":"Poelman","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Division of Metabolic Diseases and Genetics, Center for Lysosomal and Metabolic Diseases Erasmus MC University Medical Center Dr Molewaterplein 60 Rotterdam 3015 GJ The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"I.","family":"Plug","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Division of Metabolic Diseases and Genetics, Center for Lysosomal and Metabolic Diseases Erasmus MC University Medical Center Dr Molewaterplein 60 Rotterdam 3015 GJ The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M.","family":"Hoogeveen\u2010Westerveld","sequence":"additional","affiliation":[{"name":"Department of Clinical Genetics, Center for Lysosomal and Metabolic Diseases Erasmus MC University Medical Center Rotterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"N. A. M. E.","family":"van der Beek","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Division of Metabolic Diseases and Genetics, Center for Lysosomal and Metabolic Diseases Erasmus MC University Medical Center Dr Molewaterplein 60 Rotterdam 3015 GJ The Netherlands"},{"name":"Department of Neurology, Center for Lysosomal and Metabolic Diseases Erasmus MC University Medical Center Rotterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"A. J. J.","family":"Reuser","sequence":"additional","affiliation":[{"name":"Department of Clinical Genetics, Center for Lysosomal and Metabolic Diseases Erasmus MC University Medical Center Rotterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"A. 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