Fabry Disease: Multidisciplinary Evaluation After 10 Years of Treatment with Agalsidase Beta

Fabry disease is an X linked disorder of metabolism due to deficient α-galactosidase A activity. Enzyme replacement therapy (ERT) with agalsidase Beta was approved by EMA in 2001 and FDA in 2003. Patients and methods: Six patients were enrolled. Baseline data was measured for renal, cardiac, and cer...

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Publicat a:JIMD Rep
Autors principals: Juan, Politei, Hernan, Amartino, Beatriz, Schenone Andrea, Gustavo, Cabrera, Antonio, Michref, Eduardo, Tanus, Raul, Dominguez, Margarita, Larralde, Mariana, Blanco, Daniela, Gaggioli, Marina, Szlago
Format: Artigo
Idioma:Inglês
Publicat: Springer Berlin Heidelberg 2014
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC4221298/
https://ncbi.nlm.nih.gov/pubmed/24850233
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/8904_2014_310
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