Announcement: GENERATION HD1 study results published

GENERATION HD1 – This Phase 3 trial was stopped in March 2021 following a benefit–risk assessment by an independent data monitoring committee. A letter published 07/12/2023 in the New England Journal of Medicine (DOI: 10.1056/NEJMc2300400) details ad hoc analyses conducted on the week 69 data. Findings suggest that younger participants with lower disease burden may potentially benefit from tominersen. This is hypothesis is currently being tested in the Phase 2 GENERATION HD2 trial.