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Reduction of podocyte globotriaosylceramide content in adult male patients with Fabry disease with amenable GLA mutations following 6 months of migalastat treatment.
Mauer, Michael; Sokolovskiy, Alexey; Barth, Jay A; Castelli, Jeffrey P; Williams, Hadis N; Benjamin, Elfrida R; Najafian, Behzad.
Affiliation
  • Mauer M; Departments of Pediatrics and Medicine, University of Minnesota, Minneapolis, Minnesota, USA.
  • Sokolovskiy A; Department of Pathology, University of Washington, Seattle, Washington, USA.
  • Barth JA; Amicus Therapeutics, Cranbury, New Jersey, USA.
  • Castelli JP; Amicus Therapeutics, Cranbury, New Jersey, USA.
  • Williams HN; Amicus Therapeutics, Cranbury, New Jersey, USA.
  • Benjamin ER; Amicus Therapeutics, Cranbury, New Jersey, USA.
  • Najafian B; Department of Pathology, University of Washington, Seattle, Washington, USA.
J Med Genet ; 54(11): 781-786, 2017 11.
Article in En | MEDLINE | ID: mdl-28756410
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Full text: 1 Database: MEDLINE Main subject: Trihexosylceramides / Fabry Disease / 1-Deoxynojirimycin / Alpha-Galactosidase / Enzyme Inhibitors / Podocytes Limits: Adult / Humans / Male / Middle aged Language: En Year: 2017 Type: Article
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Full text: 1 Database: MEDLINE Main subject: Trihexosylceramides / Fabry Disease / 1-Deoxynojirimycin / Alpha-Galactosidase / Enzyme Inhibitors / Podocytes Limits: Adult / Humans / Male / Middle aged Language: En Year: 2017 Type: Article