• A systematic review and meta-analysis of enzyme replacement therapy in late-onset Pompe Disease 

      Dornelles, Alícia Dorneles; Junges, Ana Paula Pedroso; Pereira, Tiago da Veiga; Krug, Bárbara Côrrea; Gonçalves, Candice Beatriz Treter; Llerena Junior, Juan Clinton; Kishnani, Priya Sunil; Oliveira Junior, Haliton Alves de (2021) [Artigo de periódico]
      Pompe disease (PD) is a glycogen storage disorder caused by 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 ...