Gender, mutations and residual enzymatic activity: Investigation of predictive factors of alpha-mannosidosis phenotypic presentation and of response to velmanase alfa long term enzyme replacement therapy
Dali, Christine I., Borgwardt, Line, Lund, Allan M., Cattaneo, Federica, Ardigò, Diego, Geraci, Silvia, Muschol, Nicole, Muschol, Nicole, Amraoui, Yasmina, Tylki-Szymanska, Anna, Wijburg, Frits, Van dVolume:
123
Language:
english
Journal:
Molecular Genetics and Metabolism
DOI:
10.1016/j.ymgme.2017.12.073
Date:
February, 2018
File:
PDF, 74 KB
english, 2018