Angiotensin receptor blockade mediated amelioration of mucopolysaccharidosis type I cardiac and craniofacial pathology

Mucopolysaccharidosis type I (MPS IH) is a lysosomal storage disease (LSD) caused by inactivating mutations to the alpha-L-iduronidase (IDUA) gene. Treatment focuses on IDUA enzyme replacement and currently employed methods can be non-uniform in their efficacy particularly for the cardiac and cranio...

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Pubblicato in:J Inherit Metab Dis
Autori principali: Osborn, Mark J., Webber, Beau R., McElmurry, Ronald T., Rudser, Kyle D., DeFeo, Anthony P., Muradian, Michael, Petryk, Anna, Hallgrimsson, Benedikt, Blazar, Bruce R., Tolar, Jakub, Braunlin, Elizabeth A.
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2016
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC5335863/
https://ncbi.nlm.nih.gov/pubmed/27743312
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s10545-016-9988-z
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