Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study.
Journal of medical genetics • Vol. 54, Issue 4 • 2017
Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active α-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase.
PloS one • Vol. 10, Issue 8 • 2015
A pharmacogenetic approach to identify mutant forms of α-galactosidase A that respond to a pharmacological chaperone for Fabry disease.
Human mutation • Vol. 32, Issue 8 • 2011
Enzyme replacement therapy for Fabry disease: some answers but more questions.
Therapeutics and clinical risk management • Vol. 7 • 2011
