Effect of alglucosidase alfa dosage on survival and walking ability in patients with classic infantile Pompe disease: A multicentre observational follow-up study of the European Pompe Consortium

Authors :: Imke A.M. Ditters
Hidde H. Huidekoper
Michelle E. Kruijshaar
Dimitris Rizopoulos
Andreas Hahn
Tiziana E. Mongini
François Labarthe
Marine Tardieu
Brigitte Chabrol
Anaïs Brassier
Rossella Parini
Giancarlo Parenti
Nadine A.M.E. van der Beek
Ans T. van der Ploeg
Hannerieke van den Hout
Source :: Molecular Genetics and Metabolism. 135:S39
Publication Year :: 2022
Publisher :: Elsevier BV, 2022.

Subjects :: Endocrinology
Endocrinology, Diabetes and Metabolism
Genetics
Molecular Biology
Biochemistry

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