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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...

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Detalhes bibliográficos
Main Authors: Alfadhel, Majid, Sirrs, Sandra
Formato: Artigo
Idioma:Inglês
Publicado em: Dove Medical Press 2011
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3061846/
https://ncbi.nlm.nih.gov/pubmed/21445281
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2147/TCRM.S11987
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