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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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Bibliographic Details
Published in:Iran J Pediatr
Main Authors: Moravej, Hossein, Karamizadeh, Zohre, Paran, Maryam
Format: Artigo
Language:Inglês
Published: Kowsar 2016
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Online Access: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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