Enzyme replacement therapy improves respiratory outcomes in patients with late-onset type II glycogenosis and high ventilator dependency

Serenella Servidei, Andrea Vianello, Claudio Semplicini, Luciana Paladini, Alessandra Concas, Sabrina Ravaglia, Antonio Toscano, Tiziana Mongini, Corrado Angelini, Elena Pegoraro

Risultato della ricerca: Contributo in rivistaArticolo in rivista

18 Citazioni (Scopus)

Abstract

Type II glycogenosis (GSDII) is a rare and often fatal neuromuscular disorder caused by acid alpha-glucosidase deficiency. Although alglucosidase alfa enzyme replacement therapy (ERT) significantly improves outcomes in subjects with the infantile form, its efficacy in patients with the late-onset one is not entirely clear. The long-term efficacy of ERT in late-onset GSGII complicated by severe pulmonary impairment causing high mechanical ventilation dependency was investigated in this study.
Lingua originaleEnglish
pagine (da-a)537-544
Numero di pagine8
RivistaLung
Volume191
DOI
Stato di pubblicazionePubblicato - 2013

Keywords

  • ERT
  • Pompe
  • glycogenosis
  • respiratory outcomes

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