Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naive and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study
NEUROMUSCULAR DISORDERS. Bd. 29. H. 3. 2019 S. 167-186
Erscheinungsjahr: 2019
Publikationstyp: Zeitschriftenaufsatz
| Geprüft: | Bibliothek |
Autoren
Pena, Loren D. M. (Autor)
Barohn, Richard J. (Autor)
Byrne, Barry J. (Autor)
Desnuelle, Claude (Autor)
Goker-Alpan, Ozlem (Autor)
Ladha, Shafeeq (Autor)
Laforet, Pascal (Autor)
Mengel, Karl Eugen (Autor)
Pestronk, Alan (Autor)
Pouget, Jean (Autor)
Schoser, Benedikt (Autor)
Straub, Volker (Autor)
Trivedi, Jaya (Autor)
Van Damme, Philip (Autor)
Vissing, John (Autor)
Young, Peter (Autor)
Kacena, Katherine (Autor)
Shafi, Raheel (Autor)
Thurberg, Beth L. (Autor)
Culm-Merdek, Kerry (Autor)
van der Ploeg, Ans T. (Autor)
Klassifikation
DDC Sachgruppe:
Medizin
