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Pompe disease: what are we missing?

Pompe disease is a multisystemic metabolic disorder caused by a deficiency of lysosomal acid alpha-glucosidase (GAA) leading to progressive accumulation of lysosomal glycogen, lysosomal swelling and rupture in all tissues of the human body. Furthermore, autophagic buildup, organelle abnormalities, a...

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Detalles Bibliográficos
Publicado en:Ann Transl Med
Autor Principal: Schoser, Benedikt
Formato: Artigo
Idioma:Inglês
Publicado: AME Publishing Company 2019
Assuntos:
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC6642932/
https://ncbi.nlm.nih.gov/pubmed/31392204
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.21037/atm.2019.05.29
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