Long term clinical history of an Italian cohort of infantile onset Pompe disease treated with enzyme replacement therapy
Author:
Publisher
Springer Science and Business Media LLC
Subject
Pharmacology (medical),Genetics(clinical),General Medicine
Link
http://link.springer.com/content/pdf/10.1186/s13023-018-0771-0.pdf
Reference33 articles.
1. Kishnani PS, Nicolino M, Voit T, Rogers RC, Tsai AC-H, Waterson J, et al. Chinese hamster ovary cell-derived recombinant human α-glucosidase in infantile-onset Pompe disease. J Pediatr. 2006;149:89–97.
2. Kishnani PS, Corzo D, Nicolino M, Byrne B, Mandel H, Hwu WL, et al. Recombinant human acid α-glucosidase: major clinical benefits in infantile-onset Pompe disease. Neurology. 2007;68:99–109.
3. Kishnani P, Corzo D, Leslie N, Gruskin D, van der Ploeg A, Clancy JP, et al. Early treatment with alglucosidase alfa prolongs long-term survival of infants with Pompe disease. Pediat Res. 2009;66:329–35.
4. Nicolino M, Byrne B, Wraith JE, Leslie N, Mandel H, Freyer DR, et al. Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease. Genet Med. 2009;11:210–9.
5. Kishnani PS, Goldenberg PC, DeArmey SL, Heller J, Benjamin D, Young S, et al. Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants. Mol Genet Metab. 2010;99:26–33.
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