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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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Bibliografiske detaljer
Udgivet i:Iran J Pediatr
Main Authors: Moravej, Hossein, Karamizadeh, Zohre, Paran, Maryam
Format: Artigo
Sprog:Inglês
Udgivet: Kowsar 2016
Fag:
Online adgang: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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