ArticleJournal of child neurology2012
Friedreich ataxia clinical outcome measures: natural history evaluation in 410 participants.
Article in Journal of child neurology, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers.
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
24 citing papers in PubMed, 50 citations in OpenAlex.
- 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
- Assessing airway clearance dysfunction in Friedreich's ataxia: A focus on peak cough flow.Journal of neuromuscular diseases · 2026Article
- Longitudinal analysis shows GAA1 length and baseline clinical status as robust predictors of progression in Friedreich ataxia.Journal of neurology · 2026Article
- Unrecognized high prevalence of expanded composite repeats in Friedreich ataxia.Human molecular genetics · 2026Article
- Predictive machine learning and multimodal data to develop highly sensitive, composite biomarkers of disease progression in Friedreich ataxia.Scientific reports · 2025Article
- NAD+ precursors prolong survival and improve cardiac phenotypes in a mouse model of Friedreich's Ataxia.JCI insight · 2024Article
- CRPD frontiers in movement disorders Therapeutics: From evidence to treatment and applications: Addressing Patients' Needs in the Management of the Ataxias.Clinical parkinsonism & related disorders · 2024Article
- Retinal hypoplasia and degeneration result in vision loss in Friedreich ataxia.Annals of clinical and translational neurology · 2023Article
- Frataxin deficiency lowers lean mass and triggers the integrated stress response in skeletal muscle.JCI insight · 2022Article
- Friedreich's Ataxia related Diabetes: Epidemiology and management practices.Diabetes research and clinical practice · 2022Article
- DNA methylation in Friedreich ataxia silences expression of frataxin isoform E.Scientific reports · 2022Article
- Bone Mineral Density and Current Bone Health Screening Practices in Friedreich's Ataxia.Frontiers in neuroscience · 2022Article
- Epigenetic Heterogeneity in Friedreich Ataxia Underlies VariableFrontiers in neuroscience · 2021Article
- Predictors of loss of ambulation in Friedreich's ataxia.EClinicalMedicine · 2020Article
- Measuring peripheral nerve involvement in Friedreich's ataxia.Annals of clinical and translational neurology · 2019Article
- Impact of diabetes in the Friedreich ataxia clinical outcome measures study.Annals of clinical and translational neurology · 2017Article
- Mitochondrial iron-sulfur cluster biogenesis from molecular understanding to clinical disease.Neurosciences (Riyadh, Saudi Arabia) · 2017Review
- Degenerative Ataxias: challenges in clinical research.Annals of clinical and translational neurology · 2017Review
- Progression of Friedreich ataxia: quantitative characterization over 5 years.Annals of clinical and translational neurology · 2016Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
18 authors at 10 institutions in 2 countries.
Funding
Abstract
Friedreich ataxia is an autosomal recessive neurodegenerative disorder characterized by ataxia, dysarthria, and areflexia. The authors report the progress of a large international noninterventional cohort (n = 410), tracking the natural history of disease progression using the neurologic examination-based Friedreich Ataxia Rating Scale. The authors analyzed the rate of progression with cross-sectional analysis and longitudinal analysis over a 2-year period. The Friedreich Ataxia Rating Scale captured disease progression when used at 1 and 2 years following initial evaluation, with a lower ratio of standard deviation of change to mean change over 2 years of evaluation. However, modeling of disease progression identified substantial ceiling effects in the Friedreich Ataxia Rating Scale, suggesting this measure is most useful in subjects before maximal deficit is approached.
Indexed as
Identifiers
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.