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

LDM Pena, RJ Barohn, BJ Byrne, C Desnuelle, O Goker-Alpan, S Ladha, P Laforet, KE Mengel, A Pestronk, J Pouget, B Schoser, V Straub, J Trivedi, P van Damme, J Vissing, P Young, K Kacena, R Shafi, BL Thurberg, K Culm-MerdekAns van der Ploeg

Research output: Contribution to journalArticleAcademicpeer-review

22 Citations (Scopus)
Original languageUndefined/Unknown
Pages (from-to)167-186
Number of pages20
JournalNeuromuscular Disorders
Issue number3
Publication statusPublished - 2019

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