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Fabry Disease: Incidence of the Common Later-Onset α-Galactosidase A IVS4+919G→A Mutation in Taiwanese Newborns—Superiority of DNA-Based to Enzyme-Based Newborn Screening for Common Mutations

Fabry disease is a panethnic, X-linked, inborn error of glycosphingolipid metabolism resulting from mutations in the α-galactosidase A gene (GLA) that lead to the deficient activity of the lysosomal enzyme, α-galactosidase A (α-Gal A). Affected males with no α-Gal A activity have the early-onset cla...

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Autors principals: Chien, Yin-Hsiu, Lee, Ni-Chung, Chiang, Shu-Chuan, Desnick, Robert J, Hwu, Wuh-Liang
Format: Artigo
Idioma:Inglês
Publicat: ScholarOne 2012
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3409276/
https://ncbi.nlm.nih.gov/pubmed/22437327
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2119/molmed.2012.00002
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