A French real-world study links tofersen with slower decline in SOD1-ALS
Original source
Tofersen Treatment in SOD1-ALS: Real-World Evidence from a Retrospective Multicenter Study in France (FORSLA Study). (opens in a new tab)Compass summarised this from the study's abstract.
Study details
- Studied in
- Human
Population inferred from the title and abstract by Compass.
Related topics
A retrospective study across 19 French centers found that people with SOD1-ALS treated with tofersen had slower ALS Functional Rating Scale–Revised decline after 12 months. Their blood neurofilament light chain levels also fell. Compared with a matched historical cohort, the tofersen group had longer mean survival and lower adjusted mortality risk.
Why this matters
The findings are relevant to people with SOD1-ALS and their clinicians because they provide real-world evidence associated with slower functional decline and lower disease-related markers during tofersen treatment. However, this observational study does not prove that tofersen caused these differences, and it does not establish effects for people without SOD1 mutations or change treatment guidance by itself.
Limitations and context
This was a retrospective study of 46 treated participants, with exploratory comparison to 39 matched historical pairs. Historical controls and differences between groups can introduce bias, and ALSFRS-R may not fully capture progression. The results come from one published study and require confirmation in stronger comparative research.