Sebastiaan J.M. Gaemers

Outcomes after 18 months of eliglustat therapy in treatment‐naïve adults with <scp>G</scp>aucher disease type 1: The phase 3 ENGAGE trial

Pramod K. Mistry, Elena Lukina, Hadhami Ben Turkia et al. · American Journal of Hematology · 2017 · 96 citations · Full text

Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Oral Venglustat in Healthy Volunteers

Michel Peterschmitt, Nigel P.S. Crawford, Sebastiaan J.M. Gaemers et al. · Clinical Pharmacology in Drug Development · 2020 · 96 citations · Full text

Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy

Timothy M. Cox, Guillermo Drelichman, Renata Cravo et al. · Blood · 2017 · 90 citations · Full text

Outcomes after 8 years of eliglustat therapy for Gaucher disease type 1: Final results from the Phase 2 trial

Elena Lukina, Nora Watman, Marta Dragosky et al. · American Journal of Hematology · 2018 · 75 citations · Full text

Venglustat, an orally administered glucosylceramide synthase inhibitor: Assessment over 3 years in adult males with classic Fabry disease in an open-label phase 2 study and its extension study

Patrick Deegan, Özlem Göker-Alpan, Tarekegn Geberhiwot et al. · Molecular Genetics and Metabolism · 2022 · 46 citations · Full text