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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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| Päätekijät: | , , , , , |
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| Aineistotyyppi: | Artigo |
| Kieli: | Inglês |
| Julkaistu: |
BioMed Central
2013
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| Aiheet: | |
| Linkit: | 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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