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Infantile Pompe Disease and Enzyme Replacement Therapy

dc.contributor.authorAmdani, Shahnawaz M
dc.contributor.authorSanil, Yamuna
dc.date.accessioned2018-02-05T16:36:20Z
dc.date.available2019-01-07T18:34:38Zen
dc.date.issued2017-12
dc.identifier.citationAmdani, Shahnawaz M; Sanil, Yamuna (2017). "Infantile Pompe Disease and Enzyme Replacement Therapy." Journal of Paediatrics and Child Health 53(12): 1242-1243.
dc.identifier.issn1034-4810
dc.identifier.issn1440-1754
dc.identifier.urihttps://hdl.handle.net/2027.42/141565
dc.publisherJohn Wiley & Sons Australia, Ltd.
dc.titleInfantile Pompe Disease and Enzyme Replacement Therapy
dc.typeArticleen_US
dc.rights.robotsIndexNoFollow
dc.subject.hlbsecondlevelPediatrics
dc.subject.hlbtoplevelHealth Sciences
dc.description.peerreviewedPeer Reviewed
dc.description.bitstreamurlhttps://deepblue.lib.umich.edu/bitstream/2027.42/141565/1/jpc13733.pdf
dc.description.bitstreamurlhttps://deepblue.lib.umich.edu/bitstream/2027.42/141565/2/jpc13733_am.pdf
dc.identifier.doi10.1111/jpc.13733
dc.identifier.sourceJournal of Paediatrics and Child Health
dc.identifier.citedreferencePompe JC. Over idiopatische hypertrophie van het hart. Ned. Tijdschr. Geneeskd. 1932; 76: 304.
dc.identifier.citedreferenceAmalfitano A, Bengur AR, Morse RP et al. Recombinant human acid α‐glucosidase enzyme therapy for infantile glycogen storage disease type II: Results of a phase I/II clinical trial. Genet. Med. 2001; 3: 132 – 8.
dc.identifier.citedreferenceKishnani PS, Hwu WL, Mandel H, Nicolino M, Yong F, Corzo D; Infantile‐Onset Pompe Disease Natural History Study Group. A retrospective, multinational, multicenter study on the natural history of infantile‐onset Pompe disease. J. Pediatr. 2006; 148: 671 – 6.
dc.identifier.citedreferencevan den Hout HM, Hop W, van Diggelen OP et al. The natural course of infantile Pompe’s disease: 20 original cases compared with 133 cases from the literature. Pediatrics 2003; 112: 332 – 40.
dc.identifier.citedreferenceAnsong AK, Li JS, Nozik‐Grayck E et al. Electrocardiographic response to enzyme replacement therapy for Pompe disease. Genet. Med. 2006; 8: 297 – 301.
dc.owningcollnameInterdisciplinary and Peer-Reviewed


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