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NEO1/NEO-EXT studies: Safety and exploratory efficacy of repeat avalglucosidase alfa dosing after up to 6 years in participants with late-onset pompe disease (LOPD)

Dimachkie, MM; Barohn, RJ; Byrne, B; Goker-Alpan, O; Kishnani, PS; Ladha, S; Laforet, P; Mengel, KE; Pena, LDM; Sacconi, S; Straub, V; Trivedi, J; Van Damme, P; van der Ploeg, A; Vissing, J; Young, P; Haack, KA; Ivanina, I; Lu, XY; Schoser, B

MOLECULAR GENETICS AND METABOLISM, 2021; 132 (2): S34

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