Evidence map›Paper›PMID 40488204›Full record

ReviewFrontiers in neurology2025

Clinical and cognitive assessment in Friedreich ataxia clinical trials: a review.

Álvaro Darriba, Arnold Munnich, Pedro Cardoso-Leite, Benoît Funalot, Florian Waszak

Abstract readReview
In one paragraph

Review in Frontiers in neurology, 2025. 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. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

5 authors.

Álvaro DarribaUniversité Paris Cité, CNRS, UMR 8002 INCC, Paris, France.
Arnold MunnichInstitut Imagine, UMR 1163, Hôpital Necker and Université Paris Cité, Paris, France.
Pedro Cardoso-LeiteDepartment of Behavioural and Cognitive Sciences, University of Luxembourg, Esch-sur-Alzette, Luxembourg.
Benoît FunalotDepartment of Medical Genetics, Assistance Publique-Hôpitaux de Paris, Hôpital Necker Enfants Malades, Paris, France.
Florian WaszakUniversité Paris Cité, CNRS, UMR 8002 INCC, Paris, France.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Friedreich ataxia (FRDA) is the most common type of inherited ataxia. It is a neurodegenerative disorder characterized by progressive gait and limb ataxia, dysarthria, areflexia, and reduced proprioception and vibration sensation. Although a number of clinical trials have been conducted, there is currently no cure for this disease. In this article we review those clinical trials with a focus on the instruments used as endpoints to assess clinical progression, and discuss the potential benefits of integrating additional measures, including assessments from overlooked domains. We also review tools used to evaluate cognitive functions in individuals with FRDA, particularly those employing quantitative, objective, and time-based measures. We argue for the inclusion of cognitive and speech-related assessments in clinical trials, and examine the potential of developments in cognitive neuroscience and technology to address current measurement challenges and support more accurate and comprehensive evaluation of treatment effects. These innovations have the potential to complement existing approaches, enhance trial design, and advance clinical care.

Indexed as

behavioral assessmentclinical trialscognitive assessmentFriedreich’s ataxiaoutcome measures

Identifiers

PMID40488204
PMCPMC12142069

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

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.