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Albuterol as an adjunctive treatment to enzyme replacement therapy in infantile-onset Pompe disease()

BACKGROUND: Early initiation of enzyme replacement therapy (ERT) with recombinant human acid alpha-glucosidase is an effective treatment for patients with infantile-onset Pompe disease (IOPD) but cannot prevent a slow progression of myopathy. Albuterol has been shown to be helpful in adult patients...

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Bibliografiske detaljer
Udgivet i:Mol Genet Metab Rep
Main Authors: Chien, Yin-Hsiu, Hwu, Wuh-Liang, Lee, Ni-Chung, Tsai, Fuu-Jen, Koeberl, Dwight D., Tsai, Wen-Hui, Chiu, Pao-Chin, Chang, Chaw-Liang
Format: Artigo
Sprog:Inglês
Udgivet: Elsevier 2017
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5406275/
https://ncbi.nlm.nih.gov/pubmed/28480166
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2017.04.004
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