Molecular genetics and metabolism reports
Real-world experience of switching to taliglucerase among patients with Gaucher disease in Québec: A case series.
Angelo Rizzolo, Marie-Claude Miron, Jean-François Delisle, Nathalie Alos, Philippe M Campeau, François Mercier, Gaucher Disease Program of Québec
Published: 202510.1016/j.ymgmr.2025.101257
Abstract
Open AccessEnzyme replacement therapy (ERT) for Gaucher disease (GD) effectively prevents skeletal, visceral, and hematologic complications of this inherited, lysosomal storage disorder. Taliglucerase is one of the three commercially available ERT products and became the recommended first-line therapy in Québec, Canada in 2016. Thus, 19 patients were switched from imiglucerase to taliglucerase, but more than a quarter experienced significant side effects. Here, we summarize these patients' clinical course and describe 6 suspected product-related adverse-effects.