09/22/2026
Hope is powered by science, made possible through partnership, and driven by the determination of patients and families.
Today, we announced positive results from the Phase 3 FUSION trial evaluating ulefnersen, an investigational RNA-targeted medicine, in people living with FUS-ALS. As the first FUS-ALS clinical trial to meet its primary endpoint, these findings mark a significant milestone for the FUS-ALS community.
We are deeply grateful to the patients, carers, investigators, advocates and the broader FUS-ALS community whose courage, commitment and partnership helped make this achievement possible. Their contributions, trust and courage continue to move science forward and bring us closer to a potential targeted treatment.
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