# Tofersen Treatment in <i>SOD1</i>-ALS: Real-World Evidence from a Retrospective Multicenter Study in France (FORSLA Study)

Article · Erazo D, Hesters A, Bruneteau G, De La Cruz E, Bernard E, Grapperon AM, Le Masson G, Cintas P, Soriani MH, Guy N, Cassereau J, Camdessanché JP, Genestet S, Corcia P, Pittion S, Danel V, Filliatre M, Choumert A, Jacquin-Piques A, Salachas F, Esselin F, Bohic A, Lautrette G, Attarian S, Catteau J and 6 more

- Archive id: doi:10.1016/j.mayocpiqo.2026.100739
- Published: 2026-07-27
- Venue: Mayo Clinic proceedings. Innovations, quality & outcomes
- DOI: https://doi.org/10.1016/j.mayocpiqo.2026.100739
- PMID: 42548788
- PMCID: PMC13429897
- Citations: 1
- Source URL: https://doi.org/10.1016/j.mayocpiqo.2026.100739
- PDF: https://europepmc.org/articles/PMC13429897/pdf
- Provenance: europepmc

## Abstract

Objective To evaluate the effectiveness of Tofersen in patients with superoxide dismutase 1 gene ( SOD1-ALS ) patients in France in a real-world setting, using disease progression within patient comparisons and with a historical cohort. Patients and methods Patients with SOD1 -ALS were included from across 19 French FILSLAN network centers. Baseline was defined as the treatment initiation date. Main endpoints were the ALSFRS-R progression rate and plasmatic neurofilament light chain (NfL) levels at baseline and 12 months after baseline. Results In the Tofersen Cohort (N=46), within-group comparisons showed that the mean ALS functional rating scale revised (ALSFRS-R) progression rate slowed from 0.53 ± 0.5 at baseline to 0.22 ± 0.3 point/month at 12 months ( P =.006). NfL levels significantly decreased from 89.0 ± 9.0 pg/ml at baseline to 29.2 ± 19.5.5 at 12 months ( P =.004). Exploratory comparisons with a propensity score (PS) matched historical cohort (39 matched pairs) using a mixed-effects model, ALSFRS-R progression rate at baseline, 6 months, and 12 months after baseline, showed no statistically significant differences between groups P =.30, whereas longitudinal ALSFRS-R scores differed significantly between groups (time-treatment interaction P =.006). The mean survival of the PS matched population was longer in the Tofersen Cohort (42.6 months) than the Historical Cohort (31.8 months) P =.004. Time-dependent adjusted cox analysis showed that Tofersen was associated with a reduction in mortality risk (adjusted HR=0.34; 95% CI, 0.12-0.91; P =.03). Conclusion Tofersen seems to be associated with slower functional decline and reduced NfL levels. While limitations of retrospective design and ALSFRS-R sensitivity must be acknowledged, these findings provide real-world evidence suggesting a clinical benefit of Tofersen.

_Abstract reproduced under CC-BY (article license as recorded by Europe PMC; abstract reproduced unmodified apart from markup removal)._

Topics: [Alzheimer's, Parkinson's & Neurodegeneration](https://space-kitty.com/topics/neurodegeneration)

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