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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Udgivet i:Neurology
Main Authors: 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.
Format: Artigo
Sprog:Inglês
Udgivet: Lippincott Williams & Wilkins 2019
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6946483/
https://ncbi.nlm.nih.gov/pubmed/31619483
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1212/WNL.0000000000008441
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