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Efficacy of avalglucosidase alfa on forced vital capacity percent predicted in treatment-naïve patients with late-onset Pompe disease: A pooled analysis of clinical trials.
Mozaffar T, Riou França L, Msihid J, Shukla P, Proskorovsky I, Zhou T, Periquet M, An Haack K, Pollissard L, Straub V. Mozaffar T, et al. Among authors: riou franca l. Mol Genet Metab Rep. 2024 Jun 26;40:101109. doi: 10.1016/j.ymgmr.2024.101109. eCollection 2024 Sep. Mol Genet Metab Rep. 2024. PMID: 39035044 Free PMC article.
14 results