Improvement of gastrointestinal symptoms in a significant proportion of male patients with classic Fabry disease treated with agalsidase beta: A Fabry Registry analysis stratified by phenotype

BACKGROUND: Fabry disease is an inherited disorder of glycolipid metabolism with progressive involvement of multiple organs, including the gastrointestinal tract, in classically affected male patients. Clinical presentations in males with later-onset Fabry phenotypes are more heterogeneous and large...

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Dettagli Bibliografici
Pubblicato in:Mol Genet Metab Rep
Autori principali: Hopkin, Robert J., Feldt-Rasmussen, Ulla, Germain, Dominique P., Jovanovic, Ana, Martins, Ana Maria, Nicholls, Kathleen, Ortiz, Alberto, Politei, Juan, Ponce, Elvira, Varas, Carmen, Weidemann, Frank, Yang, Meng, Wilcox, William R.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Elsevier 2020
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Research Paper
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC7606866/
https://ncbi.nlm.nih.gov/pubmed/33163363
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2020.100670
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