Long term enzyme replacement therapy for Fabry disease: effectiveness on kidney, heart and brain

BACKGROUND: Fabry disease is an X-linked lysosomal storage disorder caused by α-galactosidase A deficiency leading to renal, cardiac, cerebrovascular disease and premature death. Treatment with α-galactosidase A (enzyme replacement therapy, ERT) stabilises disease in some patients, but long term eff...

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Autors principals: Rombach, Saskia M, Smid, Bouwien E, Bouwman, Machtelt G, Linthorst, Gabor E, Dijkgraaf, Marcel G W, Hollak, Carla E M
Format: Artigo
Idioma:Inglês
Publicat: BioMed Central 2013
Matèries:
Research
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3626869/
https://ncbi.nlm.nih.gov/pubmed/23531228
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1750-1172-8-47
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