CHINESE HAMSTER OVARY CELL-DERIVED RECOMBINANT HUMAN ACID α-GLUCOSIDASE IN INFANTILE-ONSET POMPE DISEASE

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 <1% of normal,...

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Bibliografiska uppgifter
Huvudupphovsmän: Kishnani, Priya Sunil, Nicolino, Marc, Voit, Thomas, Rogers, R. Curtis, Tsai, Anne Chun-Hui, Waterson, John, Herman, Gail E., Amalfitano, Andreas, Thurberg, Beth L., Richards, Susan, Davison, Mark, Corzo, Deyanira, Chen, YT
Materialtyp: Artigo
Språk:Inglês
Publicerad: 2006
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Article
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC2692727/
https://ncbi.nlm.nih.gov/pubmed/16860134
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.jpeds.2006.02.035
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