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Cognitive and academic outcomes in long-term survivors of infantile-onset Pompe disease: A longitudinal follow-up

This study examines the long-term cognitive and academic outcomes of 11 individuals with infantile onset Pompe disease (IOPD) (median age = 11 years, 1 month, range = 5 years, 6 months through 17 years of age) treated with enzyme replacement therapy from an early age. All participants (7 males, 4 fe...

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Detalhes bibliográficos
Publicado no:Mol Genet Metab
Main Authors: Spiridigliozzi, Gail A., Keeling, Lori A., Stefanescu, Mihaela, Li, Cindy, Austin, Stephanie, Kishnani, Priya S.
Formato: Artigo
Idioma:Inglês
Publicado em: 2017
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5985833/
https://ncbi.nlm.nih.gov/pubmed/28495044
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2017.04.014
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