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Postmortem Findings and Clinical Correlates in Individuals with Infantile-Onset Pompe Disease

Pompe disease (OMIM 232300), a glycogen storage disorder caused by deficiency in the lysosomal enzyme acid alpha-glucosidase (EC 3.2.1.20), results in weakness and cardiomyopathy in infants affected with the classic form. Although the primary disease manifestations are due to glycogen accumulation i...

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Publicat a:JIMD Rep
Autors principals: Pena, Loren D. M., Proia, Alan D., Kishnani, Priya S.
Format: Artigo
Idioma:Inglês
Publicat: Springer Berlin Heidelberg 2015
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC4484900/
https://ncbi.nlm.nih.gov/pubmed/25763511
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/8904_2015_426
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