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Mini-COMET: Individual-level treatment responses in infantile-onset Pompe disease participants receiving avalglucosidase alfa or alglucosidase alfa who previously received alglucosidase alfa

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

MOLECULAR GENETICS AND METABOLISM, 2021; 132 (): S75

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