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

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
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
Series:Orphanet Journal of Rare Diseases
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

Internet

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

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