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The effect of enzyme replacement therapy on clinical outcomes in male patients ...

Germain, DP; Elliott, PM; Falissard, B; Fomin, VV; Hilz, MJ; Jovanovic, A; Kantola, I; Linhart, A; Mignani, R; Namdar, M; Nowak, A; Oliveira, JP

Background: Enzyme replacement therapy (ERT) with recombinant human a-galactosidase has been available for the treatment of Fabry disease since 2001 in Europe and 2003 in the USA. Treatment outcomes with ERT are dependent on baseline patient characteristics, and published data are derived from heterogeneous study populations. Methods: We conducted a comprehensive systematic literature review of all original art...

Date: 2019   |   Origin: Repositório Aberto da Universidade do Porto
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