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...
| Published in: | Journal of Comparative Effectiveness Research |
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| Main Authors: | , , , , , , , , , |
| Format: | Article |
| Language: | English |
| Published: |
Becaris Publishing Limited
2024-09-01
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| Subjects: |