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

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+mi...

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Asıl Yazarlar:	Simon Shohet, Noemi Hummel, Shuai Fu, Ian Keyzor, Alasdair MacCulloch, Neil Johnson, Jeff Castelli, Ilona Czarny-Ozga, Tahseen Mozaffar, Howard Thom
Materyal Türü:	Artigo
Dil:	Inglês
Baskı/Yayın Bilgisi:	Becaris Publishing Limited 2024-09-01
Seri Bilgileri:	Journal of Comparative Effectiveness Research
Konular:	cipaglucosidase alfa indirect treatment comparison miglustat pompe disease
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