Treatment of infantile Pompe disease with alglucosidase alpha: the UK experience
Author:
Publisher
Wiley
Subject
Genetics (clinical),Genetics
Link
http://www.springerlink.com/index/pdf/10.1007/s10545-010-9206-3
Reference7 articles.
1. http://www.ncg.nhs.uk/?dl_id=77
2. Kishnani PS, Hwu WL, Mandel H, Nicolino M, Yong F, Corzo D (2006) A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease. J Pediatr 148:671–676
3. Kishnani PS, Corzo D, Nicolino M, Byrne B, Mandel H, Hwu WL, Leslie N et al (2007) Recombinant human acid α-glucosidase: Major clinical benefits in infantile-onset Pompe disease. Neurology 68:99–109
4. Kishnani PS, Corzo D, Leslie N, Gruskin D, van der Ploeg A, Clancy JP, Parini R et al (2009) Pediatr Res 66:329–335
5. Kishnani PS, Goldenburg PC, DeArmey SL, Heller J, Benjamin D, Young S, Bali D et al (2010) Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants. Mol Genet Metab 99:26–33
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