Laronidase Replacement Therapy and Left Ventricular Function in Mucopolysaccharidosis I

We assessed the effects of long-term laronidase replacement therapy (LRT) on the left ventricular (LV) function of a 52-year-old adult woman with mucopolysaccharidosis I (MPS I). The urinary uronic acid concentration significantly decreased by 78.7% (from 75 to 16 mg/g creatinine) after LRT; thereaf...

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Udgivet i:JIMD Rep
Main Authors: Harada, Haruhito, Niiyama, Hiroshi, Katoh, Atsushi, Ikeda, Hisao
Format: Artigo
Sprog:Inglês
Udgivet: Springer Berlin Heidelberg 2014
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4221302/
https://ncbi.nlm.nih.gov/pubmed/24850236
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/8904_2014_316
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