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

Authors

Loren D M Pena, Duke University
Alan Pestronk, Washington University School of Medicine in St. Louis
et al

Journal

Neuromuscular disorders

Publication Date

3-1-2019

Volume

29

Issue

3

First Page

167

Last Page

186

Document Type

Open Access Publication

DOI

10.1016/j.nmd.2018.12.004

Rights and Permissions

Pena LDM, Barohn RJ, Byrne BJ, Desnuelle C, Goker-Alpan O, Ladha S, Laforêt P, Mengel KE, Pestronk A, Pouget J, Schoser B, Straub V, Trivedi J, Van Damme P, Vissing J, Young P, Kacena K, Shafi R, Thurberg BL, Culm-Merdek K, van der Ploeg AT; NEO1 Investigator Group. 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. Neuromuscul Disord. 2019 Mar;29(3):167-186. doi.org/10.1016/j.nmd.2018.12.004. © 2018 The Authors. Published by Elsevier B.V. This is an open access article under the CC BY-NC-ND license. (http://creativecommons.org/licenses/by-nc-nd/4.0/)

Recommended Citation

Pena, Loren D M; Pestronk, Alan; and et al, "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." Neuromuscular disorders. 29, 3. 167 - 186. (2019).
https://digitalcommons.wustl.edu/open_access_pubs/11800