BIOCHEMICAL AND PHARMACOLOGICAL CHARACTERIZATION OF DIFFERENT RECOMBINANT ACID α-GLUCOSIDASE PREPARATIONS EVALUATED FOR THE TREATMENT OF POMPE DISEASE

Pompe disease results in the accumulation of lysosomal glycogen in multiple tissues due to a deficiency of acid alpha-glucosidase (GAA). Enzyme replacement therapy for Pompe disease was recently approved in Europe, the U.S., Canada and Japan using a recombinant human GAA (Myozyme, alglucosidase alfa...

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Detalhes bibliográficos
Main Authors: McVie-Wylie, A.J., Lee, K.L., Qiu, H., Jin, X., Do, H., Gotschall, R., Thurberg, B.L., Rogers, C., Raben, N., O’Callaghan, M., Canfield, W., Andrews, L., McPherson, J.M., Mattaliano, R.J.
Formato: Artigo
Idioma:Inglês
Publicado em: 2008
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2774491/
https://ncbi.nlm.nih.gov/pubmed/18538603
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2008.04.009
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