Chinese hamster ovary cell-derived recombinant human acid α-glucosidase in infantile-onset Pompe disease

Priya Sunil Kishnani, Marc Nicolino, Thomas Voit, R. Curtis Rogers, Anne Tsai, John Waterson, Gail E. Herman, Andreas Amalfitano, Beth L. Thurberg, Susan Richards, Mark Davison, Deyanira Corzo, Y. T. Chen

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216 Scopus citations

Abstract

Objective: To conduct an open-label, multinational, multicenter study examining the safety and efficacy of recombinant human acid α-glucosidase (rhGAA) in treatment of infantile-onset Pompe disease. Study design: We enrolled 8 infant patients who had Pompe disease with GAA activity

Original languageEnglish (US)
Pages (from-to)89-97
Number of pages9
JournalJournal of Pediatrics
Volume149
Issue number1
DOIs
Publication statusPublished - Jul 2006
Externally publishedYes

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ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health

Cite this

Kishnani, P. S., Nicolino, M., Voit, T., Rogers, R. C., Tsai, A., Waterson, J., ... Chen, Y. T. (2006). Chinese hamster ovary cell-derived recombinant human acid α-glucosidase in infantile-onset Pompe disease. Journal of Pediatrics, 149(1), 89-97. https://doi.org/10.1016/j.jpeds.2006.02.035