Journal article
Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study
DA Hughes, K Nicholls, SP Shankar, G Sunder-Plassmann, D Koeller, K Nedd, G Vockley, T Hamazaki, R Lachmann, T Ohashi, I Olivotto, N Sakai, P Deegan, D Dimmock, F Eyskens, DP Germain, O Goker-Alpan, E Hachulla, A Jovanovic, CM Lourenco Show all
Journal of Medical Genetics | BMJ PUBLISHING GROUP | Published : 2017
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 therapy (ERT), stabilises specific mutant (amenable) forms of α- Gal to facilitate normal lysosomal trafficking. Methods The main objective of the 18-month, randomised, active-controlled ATTRACT study was to assess the effects of migalastat on renal function in patients with Fabry disease previously treated with ERT. Effects on heart, disease substrate, patient-reported outcomes (PROs) and safety were..
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Awarded by Japan Society for the Promotion of Science
Funding Acknowledgements
Funding Amicus Therapeutics, Cranbury, NJ, USA.