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Mini-COMET study: Effects of 97 weeks of avalglucosidase alfa dosing on ptosis in participants with infantile-onset Pompe disease who were previously treated with alglucosidase alfa

Kishnani, PS; Weinert, MC; Davison, J; Broomfield, A; Chien, YH; Hahn, S; Kronn, D; Kumada, S; Ohki, H; Pichard, S; Haack, KA; Tammireddy, S; Zhou, T; Prakalapakorn, SG

MOLECULAR GENETICS AND METABOLISM, 2023; 138 (2): 74

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