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Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease
Priya S. Kishnani, Barry J. Byrne, Kristl G. Claeys, Jordi Diaz-Manera, Mazen M. Dimachkie, Hani Kushlaf, Tahseen Mozaffar, Mark Roberts, Benedikt Schoser, Noemi Hummel, Blaž Koritnik
Izvirni znanstveni članek
Oznake: Pompe disease;patient-reported outcomes;health-related quality of life;SARS-Cov-2;
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 alglucosidase alf ...
Leto: 2024 Vir: Digitalni repozitorij raziskovalnih organizacij Slovenije
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