Chaperon migalastat versus enzymová terapie u Fabryho choroby - 18měsíční studie ATTRACT : Komentář

Abstrakt

Komentář k článku: Hughes DA, Nicholls K, Shankar SP, et al. Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT.

J Med Genet doi:10.1136/jmedgenet-2016-104178.