ArticleMovement disorders : official journal of the Movement Disorder Society2010
Measuring the rate of progression in Friedreich ataxia: implications for clinical trial design.
Article in Movement disorders : official journal of the Movement Disorder Society, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 66 papers, 2 of them syntheses that pooled 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.
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.
Who cites it
66 citing papers in PubMed, 2 syntheses or guidelines pooled it, 120 citations in OpenAlex.
- Exercise and Physical Therapy Interventions for Children with Ataxia: A Systematic Review.Cerebellum (London, England) · 2019Pooled it
- Ataxia rating scales--psychometric profiles, natural history and their application in clinical trials.Cerebellum (London, England) · 2012Pooled it
- Double blind trial of a deuterated form of linoleic acid (RT001) in Friedreich ataxia.Journal of neurology · 2023Trial
- Results of a randomized double-blind study evaluating luvadaxistat in adults with Friedreich ataxia.Annals of clinical and translational neurology · 2021Trial
- Randomized, double-blind, placebo-controlled study of interferon-Annals of clinical and translational neurology · 2019Trial
- Safety, pharmacodynamics, and potential benefit of omaveloxolone in Friedreich ataxia.Annals of clinical and translational neurology · 2019Trial
- An open-label trial in Friedreich ataxia suggests clinical benefit with high-dose resveratrol, without effect on frataxin levels.Journal of neurology · 2015Trial
- Assessment of neurological efficacy of idebenone in pediatric patients with Friedreich's ataxia: data from a 6-month controlled study followed by a 12-month open-label extension study.Journal of neurology · 2012Trial
- Serial assessment of echocardiographic measures in Friedreich ataxia.American heart journal plus : cardiology research and practice · 2026Article
- Assessing airway clearance dysfunction in Friedreich's ataxia: A focus on peak cough flow.Journal of neuromuscular diseases · 2026Article
- Recent developments in Friedreich's ataxia: a state-of-the-art review.Brain communications · 2026Review
- Peripheral frataxin levels govern long-term clinical progression in Friedreich ataxia.BMJ neurology open · 2026Article
- Predictive machine learning and multimodal data to develop highly sensitive, composite biomarkers of disease progression in Friedreich ataxia.Scientific reports · 2025Article
- Measurement Properties of the Friedreich Ataxia Rating Scale in Patients with Spinocerebellar Ataxia.Neurology and therapy · 2025Article
- Content Validity of the Friedreich Ataxia Rating Scale in Patients with Spinocerebellar Ataxia.Neurology and therapy · 2025Article
- Cerebellar cognitive affective syndrome in patients with spinocerebellar ataxia type 10.PloS one · 2025Article
- At-home wearable-based monitoring predicts clinical measures and biological biomarkers of disease severity in Friedreich's Ataxia.Communications medicine · 2024Article
- Frataxin analysis using triple quadrupole mass spectrometry: application to a large heterogeneous clinical cohort.Journal of neurology · 2024Article
- Perspectives of the Friedreich ataxia community on gene therapy clinical trials.Molecular therapy. Methods & clinical development · 2024Article
- Propensity matched comparison of omaveloxolone treatment to Friedreich ataxia natural history data.Annals of clinical and translational neurology · 2024Article
6 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
12 authors at 9 institutions in 1 country.
Funding
Abstract
Friedreich ataxia is an autosomal recessive neurodegenerative disorder characterized by ataxia of all four limbs, dysarthria, and arreflexia. A variety of measures are currently used to quantify disease progression, including the Friedreich Ataxia Rating Scale, examiner-rated functional disability scales, self-reported activities of daily living and performance measures such as the timed 25-foot walk, 9-hole pegboard test, PATA speech test, and low-contrast letter acuity vision charts. This study examines the rate of disease progression over one and two years in a cohort of 236 Friedreich ataxia patients using these scales and performance measure composites. The Friedreich Ataxia Rating Scale and performance-measure composites captured disease progression, with a greater sensitivity to change over 2 years than over 1 year. The measures differed in their sensitivity to change and in possible bias. These results help to establish norms for progression in FRDA that can be useful in measuring the long-term success of therapeutic agents and defining sample-size calculations for double-blind clinical trials.
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What OpenQuestion holds
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 OpenQuestion graph.