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Fabry disease: recent advances in pathology, diagnosis, treatment and monitoring
BACKGROUND: 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 in this disorder, and has prompted a broad range of rese...
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| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
BioMed Central
2009
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| Online Zugang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC2768700/ https://ncbi.nlm.nih.gov/pubmed/19818152 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1750-1172-4-21 |
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