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

Autor:	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
Rok vydání:	2022
Předmět:	Endocrinology Endocrinology Diabetes and Metabolism Genetics Molecular Biology Biochemistry
Zdroj:	Molecular Genetics and Metabolism. 135:S39
ISSN:	1096-7192
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=doi_________::ef326804d862d7cc946fb005efec48fd https://doi.org/10.1016/j.ymgme.2021.11.087 Zobrazit plný text záznamu Full Text from ScienceDirect