Effect of alglucosidase alfa dosage on survival and walking ability in patients with classic infantile Pompe disease: a multicentre observational cohort study from the European Pompe Consortium.
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D. Rizopoulos | T. Mongini | M. Kruijshaar | G. Parenti | A. Brassier | A. T. van der Ploeg | B. Chabrol | F. Labarthe | R. Parini | A. Hahn | H. Huidekoper | M. Tardieu | J. V. D. van den Hout | N. M. van der Beek | I. Ditters