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

Research output: Contribution to journalArticle

214 Citations (Scopus)

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
StatePublished - Jul 2006
Externally publishedYes

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Glycogen Storage Disease Type II
Glucosidases
Cricetulus
Ovary
Acids
Multicenter Studies
Safety
Therapeutics
human GAA protein

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

Chinese hamster ovary cell-derived recombinant human acid α-glucosidase in infantile-onset Pompe disease. / Kishnani, Priya Sunil; Nicolino, Marc; Voit, Thomas; Rogers, R. Curtis; Tsai, Anne; Waterson, John; Herman, Gail E.; Amalfitano, Andreas; Thurberg, Beth L.; Richards, Susan; Davison, Mark; Corzo, Deyanira; Chen, Y. T.

In: Journal of Pediatrics, Vol. 149, No. 1, 07.2006, p. 89-97.

Research output: Contribution to journalArticle

Kishnani, PS, Nicolino, M, Voit, T, Rogers, RC, Tsai, A, Waterson, J, Herman, GE, Amalfitano, A, Thurberg, BL, Richards, S, Davison, M, Corzo, D & Chen, YT 2006, 'Chinese hamster ovary cell-derived recombinant human acid α-glucosidase in infantile-onset Pompe disease', Journal of Pediatrics, vol. 149, no. 1, pp. 89-97. https://doi.org/10.1016/j.jpeds.2006.02.035
Kishnani, Priya Sunil ; Nicolino, Marc ; Voit, Thomas ; Rogers, R. Curtis ; Tsai, Anne ; Waterson, John ; Herman, Gail E. ; Amalfitano, Andreas ; Thurberg, Beth L. ; Richards, Susan ; Davison, Mark ; Corzo, Deyanira ; Chen, Y. T. / Chinese hamster ovary cell-derived recombinant human acid α-glucosidase in infantile-onset Pompe disease. In: Journal of Pediatrics. 2006 ; Vol. 149, No. 1. pp. 89-97.
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