Comparing the efficacy of cipaglucosidase alfa plus miglustat with alglucosidase alfa for late-onset Pompe disease: an expanded network meta-analysis utilizing patient-level and aggregate data

Aim: Treatment options for late-onset Pompe disease (LOPD) include enzyme replacement therapy (ERT) with alglucosidase alfa (alg), cipaglucosidase alfa plus miglustat (cipa + mig) and avalglucosidase alfa. However, only one randomized controlled trial (RCT) directly compared cipa + mig and alg and...

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Asıl Yazarlar:	Shuai Fu, Noemi Hummel, Simon Shohet, Neil Johnson, Alasdair MacCulloch, Jeff Castelli, William Kerr, Brian Fox, Vera Gielen
Materyal Türü:	Artigo
Dil:	Inglês
Baskı/Yayın Bilgisi:	Becaris Publishing Limited 2026-02-01
Seri Bilgileri:	Journal of Comparative Effectiveness Research
Konular:	enzyme replacement therapy indirect treatment comparison lysosomal disorder meta-analysis metabolic myopathy multilevel network meta-regression pompe disease
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