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Fabry disease: recent advances in pathology, diagnosis, treatment and monitoring
<p>Abstract</p> <p>Background</p> <p>In Fabry disease (α-galactosidase A deficiency) accumulation of Globotriaosylceramide (Gb3) leads to progressive organ failure and premature death. The introduction of enzyme replacement therapy (ERT) was the beginning of a new era i...
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Autor principal: | |
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Formato: | Artigo |
Idioma: | Inglês |
Publicado em: |
BMC
2009-10-01
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Colecção: | Orphanet Journal of Rare Diseases |
Acesso em linha: | http://www.ojrd.com/content/4/1/21 |
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