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Switching Enzyme Replacement Therapy for Late‐Onset Pompe Disease From Alglucosidase Alfa to Cipaglucosidase Alfa Plus Miglustat: Post Hoc Effect Size Analysis of PROPEL
Kushlaf, Hani, Díaz‐Manera, Jordi, Bratkovic, Drago, Byrne, Barry J., Claeys, Kristl G., Clemens, Paula R., Dimachkie, Mazen M., Kishnani, Priya S., Laforêt, Pascal, Roberts, Mark, Schoser, Benedikt, Toscano, Antonio, Castelli, Jeff, Holdbrook, Fred, Sitaraman Das, Sheela, Goldman, Mitchell, Mozaffar, Tahseen
Published in Muscle & nerve (01.08.2025)
Published in Muscle & nerve (01.08.2025)
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Journal Article
657P Cipaglucosidase alfa + miglustat in late-onset Pompe disease: two non-ambulatory patients switching from high-dose, high-frequency alglucosidase alfa
Byrne, B., Castelli, J., Jain, V., Sitaraman Das, S., Zhang, J.
Published in Neuromuscular disorders : NMD (01.10.2024)
Published in Neuromuscular disorders : NMD (01.10.2024)
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Journal Article
P373 Effect size analysis of cipaglucosidase alfa + miglustat versus alglucosidase alfa in ERT-experienced adults with late-onset Pompe disease in PROPEL
Diaz-Manera, J., Bratkovic, D., Byrne, B., Claeys, K., Dimachkie, M., Kushlaf, H., Kishnani, P., Laforêt, P., Mozaffar, T., Roberts, M., Toscano, A., Castelli, J., Raza, S., Holdbrook, F., Das, S Sitaraman, Wasfi, Y., Schoser, B.
Published in Neuromuscular disorders : NMD (01.10.2023)
Published in Neuromuscular disorders : NMD (01.10.2023)
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Journal Article
Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease
Kishnani, Priya S., Byrne, Barry J., Claeys, Kristl G., Díaz-Manera, Jordi, Dimachkie, Mazen M., Kushlaf, Hani, Mozaffar, Tahseen, Roberts, Mark, Schoser, Benedikt, Hummel, Noemi, Kopiec, Agnieszka, Holdbrook, Fred, Shohet, Simon, Toscano, Antonio
Published in Journal of patient-reported outcomes (13.11.2024)
Published in Journal of patient-reported outcomes (13.11.2024)
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Journal Article