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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

By Loren D M Pena, Richard J Barohn, Barry J Byrne, Claude Desnuelle, Ozlem Goker-Alpan, Shafeeq Ladha, Pascal Laforêt, Karl Eugen Mengel, Alan Pestronk, Jean Pouget, Benedikt Schoser, Volker Straub, Jaya Trivedi, Philip Van Damme, John Vissing, Peter Young, Katherine Kacena, Raheel Shafi, Beth L Thurberg, Kerry Culm-Merdek and Ans T van der Ploeg
Topics: /dk/atira/pure/researchoutput/pubmedpublicationtype/D016428, Journal Article, /dk/atira/pure/researchoutput/pubmedpublicationtype/D013485, Research Support, Non-U.S. Gov't, /dk/atira/pure/researchoutput/pubmedpublicationtype/D017426, Clinical Trial, Phase I, /dk/atira/pure/researchoutput/pubmedpublicationtype/D016448, Multicenter Study
Publisher: 'Elsevier BV'
Year: 2019
DOI identifier: 10.1016/j.nmd.2018.12.004
OAI identifier: oai:pure.atira.dk:publications/42a77ce9-f0b8-432b-b799-465d443f7ca2

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