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

Abstract 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...

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Bibliographic Details
Published in:Orphanet Journal of Rare Diseases
Main Authors: Jan C. van der Meijden, Michelle E. Kruijshaar, Dimitris Rizopoulos, Pieter A. van Doorn, Nadine A. M. E. van der Beek, Ans T. van der Ploeg
Format: Article
Language:English
Published: BMC 2018-05-01
Subjects:
Pompe disease
Enzyme replacement therapy
ERT
Wheelchair
Respiratory support
Hazard ratio
Online Access:http://link.springer.com/article/10.1186/s13023-018-0824-4

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http://link.springer.com/article/10.1186/s13023-018-0824-4

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