Enzyme replacement therapy reduces the risk for wheelchair dependency in adult Pompe patients

BACKGROUND: Pompe disease is a rare metabolic myopathy. In adult patients, progressive weakness of limb-girdle and respiratory muscles often leads to wheelchair and respirator dependency. Clinical studies have shown enzyme replacement therapy (ERT) to positively affect motor and respiratory outcomes...

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Bibliografiske detaljer
Udgivet i:Orphanet J Rare Dis
Main Authors: van der Meijden, Jan C., Kruijshaar, Michelle E., Rizopoulos, Dimitris, van Doorn, Pieter A., van der Beek, Nadine A. M. E., van der Ploeg, Ans T.
Format: Artigo
Sprog:Inglês
Udgivet: BioMed Central 2018
Fag:
Research
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5964954/
https://ncbi.nlm.nih.gov/pubmed/29788986
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-018-0824-4
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