Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study
Titel:
Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study
Auteur:
Pena, Loren D.M. Barohn, Richard J. Byrne, Barry J. Desnuelle, Claude Goker-Alpan, Ozlem Ladha, Shafeeq Laforêt, Pascal Mengel, Karl Eugen Pestronk, Alan Pouget, Jean Schoser, Benedikt Straub, Volker Trivedi, Jaya Van Damme, Philip Vissing, John Young, Peter Kacena, Katherine Shafi, Raheel Thurberg, Beth L. Culm-Merdek, Kerry van der Ploeg, Ans T.