Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study

Hughes, Derralynn; Nicholls, Kathy; P. Shankar, Suma; Sunder–Plassmann, Gere; M. Koeller, David; Nedd, Khan; Vockley, Gerard; Hamazaki, Takashi; Lachmann, Robin; Ohashi, Toya; Olivotto, Iacopo; Sakai, Norio; Deegan, Patrick; Dimmock, David; Eyskens, François; P. Germain, Dominique; Göker-Alpan, Özlem; Hachulla, É.; Jovanović, Ana; Marques Lourenço, Charles; Narita, Ichiei; Thomas, Mark; R. Wilcox, William; G. Bichet, Daniel; Schiffmann, Raphael; Ludington, Elizabeth; Viereck, Christopher; Kirk, John; Yu, Julie; K. Johnson, Franklin; Boudes, Pol; R. Benjamin, Elfrida; J. Lockhart, David; Barlow, Carrolee; Skuban, Nina; P. Castelli, Jeffrey; Barth, Jay; Feldt‐Rasmussen, Ulla

doi:10.1136/jmedgenet-2016-104178

Public

Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study

Published in Journal of Medical Genetics • Nov 10, 2016

Authors:

Derralynn Hughes

Kathy Nicholls

Suma P. Shankar

Abstract

Background Fabry disease is an X-linked lysosomal storage disorder caused by GLA mutations, resulting in α-galactosidase (α-Gal) deficiency and accumulation of lysosomal substrates. Migalastat, an oral pharmacological chaperone being developed as an alternative to intravenous enzyme replacement ther...

Subject

Fabry disease

Enzyme replacement therapy

Medicine

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