Coformulation of a Novel Human α-Galactosidase A With the Pharmacological Chaperone AT1001 Leads to Improved Substrate Reduction in Fabry Mice

Fabry disease is an X-linked lysosomal storage disorder caused by mutations in the gene that encodes α-galactosidase A and is characterized by pathological accumulation of globotriaosylceramide and globotriaosylsphingosine. Earlier, the authors demonstrated that oral coadministration of the pharmaco...

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書誌詳細
出版年:Mol Ther
主要な著者: Xu, Su, Lun, Yi, Brignol, Nastry, Hamler, Rick, Schilling, Adriane, Frascella, Michelle, Sullivan, Sean, Boyd, Robert E, Chang, Kate, Soska, Rebecca, Garcia, Anadina, Feng, Jessie, Yasukawa, Hidehito, Shardlow, Carole, Churchill, Alison, Ketkar, Amol, Robertson, Nicola, Miyamoto, Masahito, Mihara, Kazutoshi, Benjamin, Elfrida R, Lockhart, David J, Hirato, Tohru, Fowles, Susie, Valenzano, Kenneth J, Khanna, Richie
フォーマット: Artigo
言語:Inglês
出版事項: Nature Publishing Group 2015
主題:
Original Article
オンライン･アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC4817779/
https://ncbi.nlm.nih.gov/pubmed/25915924
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2015.87
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