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β2 Agonists Enhance the Efficacy of Simultaneous Enzyme Replacement Therapy in Murine Pompe Disease

Enzyme replacement therapy (ERT) with recombinant human acid α-glucosidase (rhGAA) has improved clinical outcomes in patients with Pompe disease; however, the response of skeletal muscle and the central nervous system to ERT has been attenuated. The poor response of skeletal muscle to ERT has been a...

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Autors principals: Koeberl, Dwight D., Li, Songtao, Dai, Jian, Thurberg, Beth L., Bali, Deeksha, Kishnani, Priya S.
Format: Artigo
Idioma:Inglês
Publicat: 2011
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3264842/
https://ncbi.nlm.nih.gov/pubmed/22154081
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2011.11.005
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