Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease

Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease

Abstract Background Late-onset Pompe disease (LOPD), a rare autosomal recessive multisystemic disorder, substantially impacts patients’ day-to-day activities, outcomes, and health-related quality of life (HRQoL). The PROPEL trial compared cipaglucosidase alfa plus miglustat (cipa+mig) with alglucosi...

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Bibliographic Details
Main Authors: Priya S. Kishnani, Barry J. Byrne, Kristl G. Claeys, Jordi Díaz-Manera, Mazen M. Dimachkie, Hani Kushlaf, Tahseen Mozaffar, Mark Roberts, Benedikt Schoser, Noemi Hummel, Agnieszka Kopiec, Fred Holdbrook, Simon Shohet, Antonio Toscano, on behalf of the PROPEL Study Group
Format: Article
Language:English
Published: SpringerOpen 2024-11-01
Series:Journal of Patient-Reported Outcomes
Subjects:
Pompe disease
Patient-reported outcomes
Patient-reported Outcome Measurement Information System
Health-related quality of life
Online Access:https://doi.org/10.1186/s41687-024-00805-w

Internet

https://doi.org/10.1186/s41687-024-00805-w

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