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The Outcome of Infantile Onset Pompe Disease in South of Iran

BACKGROUND: Infantile Onset Pompe Disease (IOPD) is a rare autosomal recessive neuromuscular disorder. It is associated with cardiomegaly, hypotonia, paresis, and death in the first year of life. Since 2006, following the use of Alglucosidase alfa as Enzyme Replacement Therapy (ERT), the patients’ s...

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Detalhes bibliográficos
Publicado no:Iran J Pediatr
Main Authors: Moravej, Hossein, Karamizadeh, Zohre, Paran, Maryam
Formato: Artigo
Idioma:Inglês
Publicado em: Kowsar 2016
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4733296/
https://ncbi.nlm.nih.gov/pubmed/26848380
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.5812/ijp.4473
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