Evidence map›Paper›PMID 42548788›Full record

ArticleMayo Clinic proceedings. Innovations, quality & outcomes2026

Tofersen Treatment in

Daniells Erazo, Adele Hesters, Gaëlle Bruneteau, Elisa De La Cruz, Emilien Bernard, Aude-Marie Grapperon, Gwendal Le Masson, Pascal Cintas, Marie-Hélène Soriani, Nathalie Guy and 21 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Mayo Clinic proceedings. Innovations, quality & outcomes, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

31 authors.

Daniells ErazoFILSLAN - French rare disease healthcare network, France.
Adele HestersFILSLAN - French rare disease healthcare network, France.
Gaëlle BruneteauFILSLAN - French rare disease healthcare network, France.
Elisa De La CruzFILSLAN - French rare disease healthcare network, France.
Emilien BernardALS and other motor neuron disease reference center, Hospices Civils de Lyon, France.
Aude-Marie GrapperonALS and other motor neuron disease reference center, Euro-NMD ERN, Assistance Publique - Hôpitaux de Marseille, Timone University Hospital, Marseille, France.
Gwendal Le MassonALS and other motor neuron disease reference center, University Hospital Bordeaux, France.
Pascal CintasFILSLAN - French rare disease healthcare network, France.
Marie-Hélène SorianiFILSLAN - French rare disease healthcare network, France.
Nathalie GuyFILSLAN - French rare disease healthcare network, France.
Julien CassereauFILSLAN - French rare disease healthcare network, France.
Jean-Philippe CamdessanchéFILSLAN - French rare disease healthcare network, France.
Steeve GenestetFILSLAN - French rare disease healthcare network, France.
Philippe CorciaFILSLAN - French rare disease healthcare network, France.
Sophie PittionFILSLAN - French rare disease healthcare network, France.
Véronique DanelFILSLAN - French rare disease healthcare network, France.
Mathilde FilliatreFILSLAN - French rare disease healthcare network, France.
Ariane ChoumertFILSLAN - French rare disease healthcare network, France.
Agnes Jacquin-PiquesFILSLAN - French rare disease healthcare network, France.
François SalachasFILSLAN - French rare disease healthcare network, France.
Florence EsselinFILSLAN - French rare disease healthcare network, France.
Adrien BohicFILSLAN - French rare disease healthcare network, France.
Géraldine LautretteFILSLAN - French rare disease healthcare network, France.
Shahram AttarianFILSLAN - French rare disease healthcare network, France.
Julie CatteauFILSLAN - French rare disease healthcare network, France.
Claire GuissartFILSLAN - French rare disease healthcare network, France.
Marie-Claire VincentFILSLAN - French rare disease healthcare network, France.
Kevin MouzatFILSLAN - French rare disease healthcare network, France.
Marie TempleFILSLAN - French rare disease healthcare network, France.
Patrick Vourc'hFILSLAN - French rare disease healthcare network, France.
Philippe CouratierFILSLAN - French rare disease healthcare network, France.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Objective: To evaluate the effectiveness of Tofersen in patients with superoxide dismutase 1 gene ( Patients and Methods: Patients with 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 ( 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.

Identifiers

PMID42548788
PMCPMC13429897

What OpenQuestion holds

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.