Treatment of Fabry’s Disease with the Pharmacologic Chaperone Migalastat
Treatment of Fabry’s Disease with the Pharmacologic Chaperone Migalastat
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Author / Creator
Germain, Dominique P , Hughes, Derralynn A , Nicholls, Kathleen , Bichet, Daniel G , Giugliani, Roberto , Wilcox, William R , Feliciani, Claudio , Shankar, Suma P , Ezgu, Fatih , Amartino, Hernan , Bratkovic, Drago , Feldt-Rasmussen, Ulla , Nedd, Khan , Sharaf El Din, Usama , Lourenco, Charles M , Banikazemi, Maryam , Charrow, Joel , Dasouki, Majed , Finegold, David , Giraldo, Pilar , Goker-Alpan, Ozlem , Longo, Nicola , Scott, C. Ronald , Torra, Roser , Tuffaha, Ahmad , Jovanovic, Ana , Waldek, Stephen , Packman, Seymour , Ludington, Elizabeth , Viereck, Christopher , Kirk, John , Yu, Julie , Benjamin, Elfrida R , Johnson, Franklin , Lockhart, David J , Skuban, Nina , Castelli, Jeff , Barth, Jay , Barlow, Carrolee and Schiffmann, Raphael
Publisher
United States: Massachusetts Medical Society
Journal title
Language
English
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Publication information
Publisher
United States: Massachusetts Medical Society
Subjects
More information
Scope and Contents
Contents
Migalastat stabilizes mutant α-galactosidase in Fabry's disease, reducing globotriaosylceramide deposition. In this study, the percentage of patients with a decrease of 50% or more in kidney interstitial capillary deposition at 6 months was similar in the migalastat and placebo groups.
Fabry’s disease is a rare, progressive, and devastating X-li...
Alternative Titles
Full title
Treatment of Fabry’s Disease with the Pharmacologic Chaperone Migalastat
Authors, Artists and Contributors
Author / Creator
Hughes, Derralynn A
Nicholls, Kathleen
Bichet, Daniel G
Giugliani, Roberto
Wilcox, William R
Feliciani, Claudio
Shankar, Suma P
Ezgu, Fatih
Amartino, Hernan
Bratkovic, Drago
Feldt-Rasmussen, Ulla
Nedd, Khan
Sharaf El Din, Usama
Lourenco, Charles M
Banikazemi, Maryam
Charrow, Joel
Dasouki, Majed
Finegold, David
Giraldo, Pilar
Goker-Alpan, Ozlem
Longo, Nicola
Scott, C. Ronald
Torra, Roser
Tuffaha, Ahmad
Jovanovic, Ana
Waldek, Stephen
Packman, Seymour
Ludington, Elizabeth
Viereck, Christopher
Kirk, John
Yu, Julie
Benjamin, Elfrida R
Johnson, Franklin
Lockhart, David J
Skuban, Nina
Castelli, Jeff
Barth, Jay
Barlow, Carrolee
Schiffmann, Raphael
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_proquest_miscellaneous_1811291407
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_1811291407
Other Identifiers
ISSN
0028-4793
E-ISSN
1533-4406
DOI
10.1056/NEJMoa1510198
