Are Anti-rhGAA Antibodies a Determinant of Treatment Outcome in Adults with Late-Onset Pompe Disease? A Systematic Review

Background: Pompe disease is a lysosomal storage disease characterised by skeletal and respiratory muscle weakness. Since 2006, enzyme replacement therapy (ERT) with alglucosidase alfa has been available. ERT significantly improves the prognosis of patients with Pompe disease. The effect of high ant...

Full description

Bibliographic Details

Published in:	Biomolecules
Main Authors:	Imke A. M. Ditters, Harmke A. van Kooten, Nadine A. M. E. van der Beek, Ans T. van der Ploeg, Hidde H. Huidekoper, Johanna M. P. van den Hout
Format:	Article
Language:	English
Published:	MDPI AG 2023-09-01
Subjects:	late-onset Pompe disease anti-rhGAA antibodies enzyme replacement therapy systematic review
Online Access:	https://www.mdpi.com/2218-273X/13/9/1414