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Enhanced Efficacy of Enzyme Replacement Therapy in Pompe Disease Through Mannose-6-Phosphate Receptor Expression in Skeletal Muscle

Enzyme replacement therapy (ERT) with acid α-glucosidase has become available for Pompe disease; however, the response of skeletal muscle, as opposed to the heart, has been attenuated. The poor response of skeletal muscle has been attributed to the low abundance of the cation-independent mannose-6-p...

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Detalhes bibliográficos
Main Authors: Koeberl, Dwight D., Luo, Xiaoyan, Sun, Baodong, McVie-Wylie, Alison, Dai, Jian, Li, Songtao, Banugaria, Suhrad G., Chen, Y-T, Bali, Deeksha S.
Formato: Artigo
Idioma:Inglês
Publicado em: 2011
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3101281/
https://ncbi.nlm.nih.gov/pubmed/21397538
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2011.02.006
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