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

ABSTRACT: 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 replacement therapy (ERT) is the primary treatment for Pompe disease. A systematic review and network meta-analysis of published evidence on the clinical effectiveness of ERT and best supportive care (BSC) was undertaken to establish the relative effectiveness of ERT compared to BSC (in the absence of ERT). Bibliographic databases were searched to identify randomised controlled trials (RCTs) or any other prospective ERT studies in patients with Pompe disease. Network meta-analyses (NMA) of RCTs were undertaken to estima

SUBMITTER: Corbett M 

PROVIDER: S-EPMC12372379 | biostudies-literature | 2025 Aug

REPOSITORIES: biostudies-literature