ReviewJournal of child neurology2012
Therapeutic developments in Friedreich ataxia.
Review 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 19 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
19 citing papers in PubMed, 34 citations in OpenAlex.
- Iron Metabolism in Aging and Age-Related Diseases.International journal of molecular sciences · 2022Review
- Outlining the Complex Pathway of Mammalian Fe-S Cluster Biogenesis.Trends in biochemical sciences · 2020Review
- Evaluation of antibodies for western blot analysis of frataxin protein isoforms.Journal of immunological methods · 2019Article
- Cysteine desulfurase is regulated by phosphorylation of Nfs1 in yeast mitochondria.Mitochondrion · 2018Article
- Liquid Chromatography-High Resolution Mass Spectrometry Analysis of Platelet Frataxin as a Protein Biomarker for the Rare Disease Friedreich's Ataxia.Analytical chemistry · 2018Article
- Review
- E3 Ligase RNF126 Directly Ubiquitinates Frataxin, Promoting Its Degradation: Identification of a Potential Therapeutic Target for Friedreich Ataxia.Cell reports · 2017Article
- Mitochondria-penetrating peptides conjugated to desferrioxamine as chelators for mitochondrial labile iron.PloS one · 2017Article
- Time-resolved functional analysis of acute impairment of frataxin expression in an inducible cell model of Friedreich ataxia.Biology open · 2016Article
- Oxidative stress in inherited mitochondrial diseases.Free radical biology & medicine · 2015Review
- Transition of Thalassaemia and Friedreich ataxia from fatal to chronic diseases.World journal of methodology · 2014Review
- Prevention and reversal of severe mitochondrial cardiomyopathy by gene therapy in a mouse model of Friedreich's ataxia.Nature medicine · 2014Article
- Cerebellar pathology in Friedreich's ataxia: atrophied dentate nuclei with normal iron content.NeuroImage. Clinical · 2014Article
- Mitochondrial iron-sulfur cluster dysfunction in neurodegenerative disease.Frontiers in pharmacology · 2014Review
- A GAA repeat expansion reporter model of Friedreich's ataxia recapitulates the genomic context and allows rapid screening of therapeutic compounds.Human molecular genetics · 2013Article
- Routine clinical testing underestimates proprioceptive deficits in Friedreich's ataxia.Cerebellum (London, England) · 2013Article
- High-throughput immunoassay for the biochemical diagnosis of Friedreich ataxia in dried blood spots and whole blood.Clinical chemistry · 2013Article
- Iron-sulfur cluster synthesis, iron homeostasis and oxidative stress in Friedreich ataxia.Molecular and cellular neurosciences · 2013Review
- Friedreich's ataxia, frataxin, PIP5K1B: echo of a distant fracas.Oxidative medicine and cellular longevity · 2013Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
1 author at 1 institution in 1 country.
Funding
Abstract
Friedreich ataxia is an inherited, severe, progressive neuro- and cardiodegenerative disorder for which there currently is no approved therapy. Friedreich ataxia is caused by the decreased expression and/or function of frataxin, a mitochondrial matrix protein that binds iron and is involved in the formation of iron-sulfur clusters. Decreased frataxin function leads to decreased iron-sulfur cluster formation, mitochondrial iron accumulation, cytosolic iron depletion, oxidative stress, and mitochondrial dysfunction. Cloning of the disease gene for Friedreich ataxia and elucidation of many aspects of the biochemical defects underlying the disorder have led to several major therapeutic initiatives aimed at increasing frataxin expression, reversing mitochondrial iron accumulation, and alleviating oxidative stress. These initiatives are in preclinical and clinical development and are reviewed herein.
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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.