Enzyme replacement therapy for the treatment of late onset Pompe disease: A systematic review and network meta-analysis

Enzyme replacement therapy for the treatment of late onset Pompe disease: A systematic review and network meta-analysis

Abstract Background Late-onset Pompe disease (LOPD) is a rare inherited genetic condition caused by deficiency of acid α-glucosidase (GAA) and accumulation of lysosomal glycogen. LOPD causes progressive muscle dysfunction and damage, leading to significant morbidity and early mortality. Enzyme repla...

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Bibliographic Details
Main Authors: Mark Corbett, Chinyereugo Umemneku-Chikere, Sarah Nevitt, Nyanar Jasmine Deng, Matthew Walton, Helen Fulbright, Chong Yew Tan, Robin Lachmann, Rachel Churchill, Robert Hodgson
Format: Article
Language:English
Published: BMC 2025-08-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Glycogen storage disease type II
Enzyme replacement therapy
Network Meta-Analysis
Humans
Online Access:https://doi.org/10.1186/s13023-025-03981-0
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