Comparing the efficacy of cipaglucosidase alfa plus miglustat with other enzyme replacement therapies for late-onset Pompe disease: a network meta-analysis utilizing patient-level and aggregate data

• Main Authors: Simon Shohet (Author), Noemi Hummel (Author), Shuai Fu (Author), Ian Keyzor (Author), Alasdair MacCulloch (Author), Neil Johnson (Author), Jeff Castelli (Author), Ilona Czarny-Ozga (Author), Tahseen Mozaffar (Author), Howard Thom (Author)
• Format: Book
• Published: Becaris Publishing Limited, 2024-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Aim: Late-onset Pompe disease is characterized by progressive loss of muscular and respiratory function. Until recently, standard of care was enzyme replacement therapy (ERT) with alglucosidase alfa. Secondgeneration ERTs avalglucosidase alfa (aval) and cipaglucosidase alfa with miglustat (cipa+mig)...