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Enzyme replacement therapy for Fabry disease: some answers but more questions
Fabry disease (FD) is a multisystem, X-linked disorder of glycosphingolipid metabolism caused by enzyme deficiency of α-galactosidase A. Affected patients have symptoms including acroparesthesias, angiokeratomas, and hypohidrosis. More serious manifestations include debilitating pain and gastrointes...
| Autores principales: | , |
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| Formato: | Texto |
| Lenguaje: | English |
| Publicado: |
Dove Medical Press
2011
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3061846/ https://www.ncbi.nlm.nih.gov/pubmed/21445281 http://dx.doi.org/10.2147/TCRM.S11987 |