Effects of enzyme replacement therapy on pain and health related quality of life in patients with Fabry disease: data from FOS (Fabry Outcome Survey)

Background: Fabry disease is an X linked lysosomal storage disease caused by deficiency of the lysosomal enzyme α-galactosidase A. This leads to accumulation of globotriaosylceramide in nearly all tissues, including the blood vessels, kidney, myocardium, and nervous system. Symptoms often begin in c...

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Bibliografiset tiedot
Päätekijät: Hoffmann, B, d Garcia, Mehta, A, Beck, M, Widmer, U, Ricci, R, on, b
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: BMJ Group 2005
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Original Article
Linkit:https://ncbi.nlm.nih.gov/pmc/articles/PMC1736022/
https://ncbi.nlm.nih.gov/pubmed/15744039
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1136/jmg.2004.025791
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