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Large variation in effects during 10 years of enzyme therapy in adults with Pompe disease

OBJECTIVE: To determine the effects of 10 years of enzyme replacement therapy (ERT) in adult patients with Pompe disease, focusing on individual variability in treatment response. METHODS: In this prospective, multicenter cohort study, we studied 30 patients from the Netherlands and France who had s...

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Detalles Bibliográficos
Autores principales:	Harlaar, Laurike, Hogrel, Jean-Yves, Perniconi, Barbara, Kruijshaar, Michelle E., Rizopoulos, Dimitris, Taouagh, Nadjib, Canal, Aurélie, Brusse, Esther, van Doorn, Pieter A., van der Ploeg, Ans T., Laforêt, Pascal, van der Beek, Nadine A. M. E.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Lippincott Williams & Wilkins 2019
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6946483/ https://www.ncbi.nlm.nih.gov/pubmed/31619483 http://dx.doi.org/10.1212/WNL.0000000000008441

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