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

• Κύριοι συγγραφείς: A. D. Dornelles (Συγγραφέας), A. P. P. Junges (Συγγραφέας), B. Krug (Συγγραφέας), C. Gonçalves (Συγγραφέας), H. A. de Oliveira Junior (Συγγραφέας), I. V. D. Schwartz (Συγγραφέας)
• Μορφή: Βιβλίο
• Έκδοση: Frontiers Media S.A., 2024-02-01T00:00:00Z.
• Θέματα: article
• Διαθέσιμο Online: 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...