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Mini-COMET: effects of avalglucosidase alfa on ptosis in participants with infantile-onset Pompe disease previously treated with alglucosidase alfa

Prakalapakorn, SG; Davison, J; Broomfield, A; Kronn, D; Labarthe, F; Brassier, A; Hahn, SH; Kumada, S; Ohki, H; Pichard, S; Haack, KA; Johnson, J; Meng, XZ; Chien, YH; Kishnani, P

MOLECULAR GENETICS AND METABOLISM, 2021; 132 (): S137

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