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Fabry Disease p.M290I Mutation is Related to Organ Involvement: A Case Report

Fabry disease (FD) is an X-linked hereditary disease. It results from mutations in the GLA gene, leading to deficient activity of the enzyme alpha-galactosidase A (α-Gal A) and progressive accumulation of undegraded glycosphingolipids in cell lysosomes. Enzyme replacement therapy (ERT) can improve t...

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Detalhes bibliográficos
Publicado no:Cureus
Main Authors: Silva, Francisca, Pestana, Nicole, Durães, José, Guimarães Rosa, Nuno, Silva, Gil
Formato: Artigo
Idioma:Inglês
Publicado em: Cureus 2021
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC8065947/
https://ncbi.nlm.nih.gov/pubmed/33907643
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.7759/cureus.14100
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