ReviewCerebellum (London, England)2024
A Milestone in the Treatment of Ataxias: Approval of Omaveloxolone for Friedreich Ataxia.
Review in Cerebellum (London, England), 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 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
20 citing papers in PubMed, 29 citations in OpenAlex.
- Remote Online-Based Application of the Cerebellar Cognitive Affective Syndrome Scale.Cerebellum (London, England) · 2026Article
- Recent Advances in Therapy for the Neurodegenerative Disorder Ataxia-Telangiectasia.International journal of molecular sciences · 2026Review
- Exercise and smoking: health rivals revealing shared protective mechanisms in Parkinson's?NPJ Parkinson's disease · 2026Review
- The TRKB Agonist 7,8-dihydroxyflavone Alleviates DNA Damage and Apoptosis in a Neuronal Cell Model of Friedreich's Ataxia.Molecular neurobiology · 2026Article
- Targeting the Milieu in Neurodegenerative Disease: Time for "Imprecision Medicine"?Aging and disease · 2025Review
- Neuroimaging Biomarkers for Friedreich Ataxia: A Cross-Sectional Analysis of the TRACK-FA Study.Annals of neurology · 2025Observational
- Omaveloxolone, But Not Dimethyl Fumarate, Improves Cardiac Function in Friedreich's Ataxia Mice With Severe Cardiomyopathy.Journal of the American Heart Association · 2025Article
- Hereditary Ataxias in Argentina.Cerebellum (London, England) · 2025Review
- Global first-in-class drugs approved in 2023-2024: Breakthroughs and insights.Innovation (Cambridge (Mass.)) · 2025Review
- Omaveloxolone Ameliorates Cognitive Deficits by Inhibiting Apoptosis and Neuroinflammation in APP/PS1 Mice.Molecular neurobiology · 2025Article
- Effect of Media Composition and Oxygen Tension on Cellular Stress Response and Nrf2 Activation in HepG2ARE Cells.Antioxidants (Basel, Switzerland) · 2025Article
- Pulling back the mitochondria's iron curtain.npj metabolic health and disease · 2025Review
- Gait characteristics in people with Friedreich ataxia: daily life versus clinic measures.Frontiers in neurology · 2025Article
- Computational drug repurposing in Parkinson's disease: Omaveloxolone and cyproheptadine as promising therapeutic candidates.Frontiers in pharmacology · 2025Article
- New and Emerging Drug and Gene Therapies for Friedreich Ataxia.CNS drugs · 2024Review
- The Triterpenoid CDDO-Methyl Ester Reduces Tumor Burden, Reprograms the Immune Microenvironment, and Protects from Chemotherapy-Induced Toxicity in a Preclinical Mouse Model of Established Lung Cancer.Antioxidants (Basel, Switzerland) · 2024Article
- Evaluating mFARS in pediatric Friedreich's ataxia: Insights from the FACHILD study.Annals of clinical and translational neurology · 2024Article
- Accelerometer-based measures in Friedreich ataxia: a longitudinal study on real-life activity.Frontiers in pharmacology · 2024Article
- Butyrate prevents visceral adipose tissue inflammation and metabolic alterations in a Friedreich's ataxia mouse model.iScience · 2023Article
- Effectiveness of rehabilitation intervention in persons with Friedreich ataxia.Frontiers in neurology · 2023Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The exciting news about the US FDA approval of omaveloxolone as the first-ever drug to be approved for an inherited ataxia is welcome news for patients and families that deal with this devastating disease as well as for health care providers and investigators with an interest in this and other rare diseases. This event is the culmination of long and fruitful collaboration between patients, their families, clinicians, laboratory researchers, patient advocacy organizations, industry, and regulatory agencies. The process has generated intense discussion about outcome measures, biomarkers, trial design, and the nature of approval process for such diseases. It also has brought hope and enthusiasm for increasingly better therapies for genetic diseases in general.
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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.