Pulmonary Outcome Measures in Long Term Survivors of Infantile Pompe Disease on Enzyme Replacement Therapy: A case series

OBJECTIVES: To report the respiratory function of school aged children with infantile Pompe disease (IPD) who started enzyme replacement therapy (ERT) in infancy and early childhood. STUDY DESIGN: This is a retrospective chart review of pulmonary function tests (PFT) of: 1) IPD patients 5 to 18 year...

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Publicado en:Pediatr Pulmonol
Main Authors: ElMallah, Mai K., Desai, Ankit K., Nading, Erica Burner, DeArmey, Stephanie, Kravitz, Richard M., Kishnani, Priya S.
Formato: Artigo
Idioma:Inglês
Publicado: 2020
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Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC7053514/
https://ncbi.nlm.nih.gov/pubmed/31899940
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ppul.24621
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