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

• Main Authors: Priya S. Kishnani (Author), Barry J. Byrne (Author), Kristl G. Claeys (Author), Jordi Díaz-Manera (Author), Mazen M. Dimachkie (Author), Hani Kushlaf (Author), Tahseen Mozaffar (Author), Mark Roberts (Author), Benedikt Schoser (Author), Noemi Hummel (Author), Agnieszka Kopiec (Author), Fred Holdbrook (Author), Simon Shohet (Author), Antonio Toscano (Author), on behalf of the PROPEL Study Group (Author)
• Format: Book
• Published: SpringerOpen, 2024-11-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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 algl...