Treatment of Fabry’s Disease with the Pharmacologic Chaperone Migalastat | Zendy

Dominique P. Germain | Zendy; Derralynn Hughes | Zendy; Kathy Nicholls | Zendy; Daniel G. Bichet | Zendy; Roberto Giugliani | Zendy; William R. Wilcox | Zendy; Claudio Feliciani | Zendy; Suma P. Shankar | Zendy; Fatih Süheyl Ezgü | Zendy; Hernan Amartino | Zendy; Drago Bratkovic | Zendy; Ulla FeldtRasmussen | Zendy; Khan Nedd | Zendy; Usama Sharaf El Din | Zendy; Charles Marques Lourenço | Zendy; Maryam Banikazemi | Zendy; Joel Charrow | Zendy; Majed Dasouki | Zendy; David N. Finegold | Zendy; Pilar Giraldo | Zendy; Özlem Göker-Alpan | Zendy; Nicola Longo | Zendy; C. Ronald Scott | Zendy; Roser Torrá | Zendy; Ahmad Tuffaha | Zendy; Ana Jovanović | Zendy; Stephen Waldek | Zendy; Seymour Packman | Zendy; Elizabeth Ludington | Zendy; Christopher Viereck | Zendy; John Kirk | Zendy; Julie Yu | Zendy; Elfrida R. Benjamin | Zendy; Franklin K. Johnson | Zendy; David J. Lockhart | Zendy; Nina Skuban | Zendy; Jeff Castelli | Zendy; Jay Barth | Zendy; Carrolee Barlow | Zendy; Raphael Schiffmann | Zendy

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Treatment of Fabry’s Disease with the Pharmacologic Chaperone Migalastat

Author(s) -

Dominique P. Germain,

Derralynn Hughes,

Kathy Nicholls,

Daniel G. Bichet,

Roberto Giugliani,

William R. Wilcox,

Claudio Feliciani,

Suma P. Shankar,

Fatih Süheyl Ezgü,

Hernan Amartino,

Drago Bratkovic,

Ulla FeldtRasmussen,

Khan Nedd,

Usama Sharaf El Din,

Charles Marques Lourenço,

Maryam Banikazemi,

Joel Charrow,

Majed Dasouki,

David N. Finegold,

Pilar Giraldo,

Özlem Göker-Alpan,

Nicola Longo,

C. Ronald Scott,

Roser Torrá,

Ahmad Tuffaha,

Ana Jovanović,

Stephen Waldek,

Seymour Packman,

Elizabeth Ludington,

Christopher Viereck,

John Kirk,

Julie Yu,

Elfrida R. Benjamin,

Franklin K. Johnson,

David J. Lockhart,

Nina Skuban,

Jeff Castelli,

Jay Barth,

Carrolee Barlow,

Raphael Schiffmann

Publication year - 2016

Publication title -

new england journal of medicine

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 19.889

H-Index - 1030

eISSN - 1533-4406

pISSN - 0028-4793

DOI - 10.1056/nejmoa1510198

Subject(s) - fabry disease , medicine , globotriaosylceramide , urology , mutant , gastroenterology , disease , biochemistry , biology , gene

Fabry's disease, an X-linked disorder of lysosomal α-galactosidase deficiency, leads to substrate accumulation in multiple organs. Migalastat, an oral pharmacologic chaperone, stabilizes specific mutant forms of α-galactosidase, increasing enzyme trafficking to lysosomes.

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