The migalastat GLP-HEK assay is the gold standard for determining amenability in patients with Fabry disease
Mol Genet Metab Rep
.
2019 Jul 19:20:100494.
doi: 10.1016/j.ymgmr.2019.100494.
eCollection 2019 Sep.
Authors
Raphael Schiffmann
1
,
Daniel G Bichet
2
,
Elfrida Benjamin
3
,
Xiaoyang Wu
3
,
Roberto Giugliani
4
Affiliations
1
Institute of Metabolic Disease, Baylor Scott & White Research Institute, Dallas, TX, USA.
2
Hôpital du Sacré-Coeur, University of Montréal, Montreal, Quebec, Canada.
3
Amicus Therapeutics, Inc., Cranbury, NJ, USA.
4
Medical Genetics Service, HCPA and Department of Genetics, UFRGS, Porto Alegre, Brazil.
PMID:
31367522
PMCID:
PMC6656697
DOI:
10.1016/j.ymgmr.2019.100494
No abstract available