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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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Bibliografiska uppgifter
I publikationen:Iran J Pediatr
Huvudupphovsmän: Moravej, Hossein, Karamizadeh, Zohre, Paran, Maryam
Materialtyp: Artigo
Språk:Inglês
Publicerad: Kowsar 2016
Ämnen:
Länkar: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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