Real-life effectiveness 1 year after switching to avalglucosidase alfa in late-onset Pompe disease patients worsening on alglucosidase alfa therapy: A French cohort study.

Céline TardFrançoise BouhourMaud MichaudStéphane BeltranMaxime FournierFlorence DemurgerEmmeline LagrangeSylvain NolletSabrina SacconiJean-Baptiste NouryArmelle MagotPascal CintasDimitri RenardJoëlle Deibener-KaminskyClaire LefeuvreJean-Baptiste DavionEmmanuelle Salort-CampanaAzzeddine ArrassiNadjib TaouaghMarco Spinazzinull nullShahram AttarianPascal Laforêt

Published in: European journal of neurology (2024)

Switching to avalglucosidase alfa demonstrated varied responses in advanced LOPD patients with failing alglucosidase alfa therapy, with a general improvement in motor stabilization.

Keyphrases

late onset
replacement therapy
early onset
end stage renal disease
ejection fraction
randomized controlled trial
smoking cessation
newly diagnosed
chronic kidney disease
systematic review
prognostic factors
stem cells
patient reported outcomes
bone marrow
cell therapy