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

Conclusions
Migalastat offers promise as a first-in-class oral monotherapy alternative treatment to intravenous ERT for patients with Fabry disease and amenable mutations.

Trial registration number:
NCT00925301; Pre-results.

Source: Journal of Medical Genetics - March 22, 2017 Category: Genetics & Stem Cells Authors: Hughes, D. A., Nicholls, K., Shankar, S. P., Sunder-Plassmann, G., Koeller, D., Nedd, K., Vockley, G., Hamazaki, T., Lachmann, R., Ohashi, T., Olivotto, I., Sakai, N., Deegan, P., Dimmock, D., Eyskens, F., Germain, D. P., Goker-Alpan, O., Hachulla, E., Jo Tags: Open access, Metabolic disorders Therapeutics Source Type: research