Global treatment responder analysis demonstrates clinically...

Molecular Genetics and Metabolism 2018 / 02 Vol. 123; Iss. 2

Global treatment responder analysis demonstrates clinically relevant effect of velmanase alfa long term enzyme replacement therapy for alpha mannosidosis, in a phase III randomized placebo controlled trial

Harmatz, Paul, Cattaneo, Federica, Ardigò, Diego, Geraci, Silvia, Amraoui, Yasmina, Gil-Campos, Mercedes, De Meirleir, Linda, Muschol, Nicole, Tylki-Szymanska, Anna, Wijburg, Frits, Van den Hout, Joha
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Volume:
123
Language:
english
Journal:
Molecular Genetics and Metabolism
DOI:
10.1016/j.ymgme.2017.12.139
Date:
February, 2018
File:
PDF, 52 KB
english, 2018
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