Improvement of Fabry Disease-Related Gastrointestinal Symptoms in a Significant Proportion of Female Patients Treated with Agalsidase Beta: Data from the Fabry Registry

Fabry disease, an X-linked inherited lysosomal storage disorder, is caused by mutations in the gene encoding α-galactosidase, GLA. In patients with Fabry disease, glycosphingolipids accumulate in various cell types, triggering a range of cellular and tissue responses that result in a wide spectrum o...

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Bibliographic Details
Published in:JIMD Rep
Main Authors: Wilcox, William R., Feldt-Rasmussen, Ulla, Martins, Ana Maria, Ortiz, Alberto, Lemay, Roberta M., Jovanovic, Ana, Germain, Dominique P., Varas, Carmen, Nicholls, Katherine, Weidemann, Frank, Hopkin, Robert J.
Format: Artigo
Language:Inglês
Published: Springer Berlin Heidelberg 2017
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Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC5874211/
https://ncbi.nlm.nih.gov/pubmed/28510034
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/8904_2017_28
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