The emerging phenotype of long-term survivors with infantile Pompe disease

PURPOSE: Enzyme replacement therapy with alglucosidase alfa for infantile Pompe disease has improved survival creating new management challenges. We describe an emerging phenotype in a retrospective review of long-term survivors. METHODS: Inclusion criteria included ventilator-free status and age ≤6...

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Main Authors: Prater, Sean N., Banugaria, Suhrad G., DeArmey, Stephanie M., Botha, Eleanor G., Stege, Erin M., Case, Laura E., Jones, Harrison N., Phornphutkul, Chanika, Wang, Raymond Y., Young, Sarah P., Kishnani, Priya S.
Format: Artigo
Sprog:Inglês
Udgivet: 2012
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3947503/
https://ncbi.nlm.nih.gov/pubmed/22538254
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gim.2012.44
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