Efficacy and safety of enzyme replacement therapy with alglucosidase alfa for the treatment of patients with infantile-onset Pompe disease: a systematic review and metanalysis

• Main Authors: A. D. Dornelles (Author), A. P. P. Junges (Author), B. Krug (Author), C. Gonçalves (Author), H. A. de Oliveira Junior (Author), I. V. D. Schwartz (Author)
• Format: Book
• Published: Frontiers Media S.A., 2024-02-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

IntroductionPompe disease (PD) is a glycogen disorder caused by the deficient activity of acid alpha-glucosidase (GAA). We sought to review the latest available evidence on the safety and efficacy of recombinant human GAA enzyme replacement therapy (ERT) for infantile-onset PD (IOPD).MethodsWe syste...