Evidence map›Paper›PMID 39806095›Full record

ArticleNeurology and therapy2025

Measurement Properties of the Friedreich Ataxia Rating Scale in Patients with Spinocerebellar Ataxia.

Michele H Potashman, Evan Popoff, Lauren C Powell, Melissa Wolfe Beiner, Ainsley Mackenzie, Vlad Coric, Sub Subramony, Matthis Synofzik, Jeremy Schmahmann, Gilbert L'Italien

Registry-linked trialAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Neurology and therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03701399 (A Phase III, Long-Term, Randomized, Double-blind, Placebo-controlled Trial of Troriluzole in Adult Participants With Spinocerebellar Ataxia.), which is not on this 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.

NCT03701399 phase3active not recruitingnot on this map

A Phase III, Long-Term, Randomized, Double-blind, Placebo-controlled Trial of Troriluzole in Adult Participants With Spinocerebellar Ataxia.

TypeinterventionalSponsorBiohaven Pharmaceuticals, Inc.Ran2019 to 2026Enrolled299ConditionsSpinocerebellar Ataxias, Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3Armstroriluzole, Placebo
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

10 authors.

Michele H PotashmanBiohaven Pharmaceuticals, Inc, 215 Church Street, New Haven, CT, 06510, USA. Michele.potashman@biohavenpharma.com.ORCID http://orcid.org/0000-0001-7389-3354
Evan PopoffBroadstreet Health Economics & Outcomes Research, Vancouver, BC, Canada.ORCID http://orcid.org/0000-0002-3233-3147
Lauren C PowellBroadstreet Health Economics & Outcomes Research, Vancouver, BC, Canada.ORCID http://orcid.org/0000-0002-4329-5166
Melissa Wolfe BeinerBiohaven Pharmaceuticals, Inc, 215 Church Street, New Haven, CT, 06510, USA.
Ainsley MackenzieBiohaven Pharmaceuticals, Inc, 215 Church Street, New Haven, CT, 06510, USA.
Vlad CoricBiohaven Pharmaceuticals, Inc, 215 Church Street, New Haven, CT, 06510, USA.
Sub SubramonyDepartment of Neurology, University of Florida College of Medicine, Gainesville, FL, USA.ORCID http://orcid.org/0000-0002-8052-9240
Matthis SynofzikDivision of Translational Genomics of Neurodegenerative Diseases, Hertie Institute for Clinical Brain Research and Center of Neurology, University Tübingen, Tübingen, Germany.ORCID http://orcid.org/0000-0002-2280-7273
Jeremy SchmahmannDepartment of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, MA, USA.ORCID http://orcid.org/0000-0003-0706-5125
Gilbert L'ItalienBiohaven Pharmaceuticals, Inc, 215 Church Street, New Haven, CT, 06510, USA.ORCID http://orcid.org/0009-0009-5196-003X

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionThe Friedreich Ataxia Rating Scale-Activities of Daily Living (FARS-ADL) is a valid, highly utilized measure for assessing ADL impacts in patients with Friedreich ataxia. We provide evidence of the psychometric validity of the FARS-ADL in two cohorts of patients with spinocerebellar ataxia (SCA).

methodsUsing data from a cohort of real-world subjects with SCA (recruited at Massachusetts General Hospital [MGH]; n = 33) and a phase 3 trial of troriluzole in adults with SCA (NCT03701399 [Study 206]; n = 217), comprising a subset of patients with the SCA3 genotype (n = 89), the psychometric measurement properties and minimal change thresholds of the FARS-ADL were examined.

resultsCeiling effects for the FARS-ADL were absent within the MGH cohort while floor effects were observed for eight of nine items. Excellent internal consistency reliability was observed (α

conclusionPsychometric evaluation showed that the FARS-ADL performed well on analyses examining the reliability and validity of the measure and can detect meaningful changes in patients with SCA, including those with SCA3.

trial registrationClinicalTrials.gov identifier, NCT03701399 (Study 206).

Indexed as

Activities of daily livingOutcomes assessmentPsychometricsSpinocerebellar ataxiasValidation study

Identifiers

PMID39806095
PMCPMC11906947

What Socratic holds

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Registered trials

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 Socratic graph.