autores Miguel Albino Silva Fernandes do Gago Nuno Jorge Carvalho de Sousa Azevedo, O. Gago M Gabriel Miltenberger-Miltenyi Nuno Sousa Cunha, D. Aksoy-Aksel, A.
palavras-chave agalsidase alfa agalsidase beta enzyme replacement therapy fabry disease gene therapy mRNA migalastat moss-derived alfa galactosidase A pegunigalsidase alfa substrate reduction