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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Detalhes bibliográficos
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.
Formato: Artigo
Idioma:Inglês
Publicado em: 2012
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Article
Acesso em linha: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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