Long-term Follow-Up of 64 Children with Classical Infantile-Onset Pompe Disease Since 2004: A French Real-Life Observational Study
Marine Tardieu,Celine Cudejko,Aline Cano,Celia Hoebeke,Delphine Bernoux,Violette Goetz,Samia Pichard,Anais Brassier,Manuel Schiff,Francois Feillet,Paul Rollier,Karine Mention,Dries Dobbelaere,Alain Fouilhoux,Caroline Espil-Taris,Didier Eyer,Frederic Huet,Ulrike Walther-Louvier,Magalie Barth,Laurent Chevret,Alice Kuster,Jeremie Lefranc,Julien Neveu,Gaele Pitelet,Juliette Ropars,Francois Rivier,Agathe Roubertie,Guy Touati,Catherine Vanhulle, Emilie Tardieu,Catherine Caillaud,Roseline Froissart, Murielle Champeaux,Francois Labarthe,Brigitte Chabrol EUROPEAN JOURNAL OF NEUROLOGY（2023）
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enzyme replacement therapy,immunomodulation,infantile-onset Pompe disease,long-term outcomes
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