Real-world experience of switching to taliglucerase among patients with Gaucher disease in Québec: A case series
Enzyme 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.
Authors
- Marie‐Claude Miron (ORCID: https://orcid.org/0000-0002-6425-4785)
- Jean‐François Delisle (ORCID: https://orcid.org/0000-0001-9430-2138)
- Pascal Bédard
- Nathalie Alos (ORCID: https://orcid.org/0000-0003-1258-7665)
- Luis H. Ospina (ORCID: https://orcid.org/0000-0003-2941-6336)
- Véronique François (ORCID: https://orcid.org/0000-0002-0454-7164)
- Anne Lortie
- Denis Bérubé
- François Mercier (ORCID: https://orcid.org/0000-0001-5324-2167)
- Angelo Rizzolo
- Philippe M. Campeau (ORCID: https://orcid.org/0000-0001-9713-7107)
- Sarit Assouline
- Claude Meilleur
Institutions
- Jewish General Hospital (CA)
- Centre Hospitalier Universitaire Sainte-Justine (CA)
Publication Details
- Journal
- Molecular Genetics and Metabolism Reports
- Published
- 2025-09-25
- DOI
- https://doi.org/10.1016/j.ymgmr.2025.101257
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00