Comprehensive long-term efficacy and safety of recombinant human alpha-mannosidase (velmanase alfa) treatment in patients with alpha-mannosidosis.
Allan M Lund, Line Borgwardt, Federica Cattaneo, Diego Ardigò, Silvia Geraci, Mercedes Gil-Campos, Linda De Meirleir, Cécile Laroche, Philippe Dolhem, Duncan Cole, Anna Tylki-Szymanska, Monica Lopez-Rodriguez, Encarna Guillén-Navarro, Christine I Dali, Bénédicte Héron, Jens Fogh, Nicole Muschol, Dawn Phillips, J M Hannerieke Van den Hout, Simon A Jones, Yasmina Amraoui, Paul Harmatz, Nathalie Guffon
Journal of inherited metabolic disease 2019;41(6):1225-1233