Evidence map›Paper›PMID 26782317›Full record

ReviewNeurodegenerative disease management2016

Emerging therapies in Friedreich's ataxia.

Tanya V Aranca, Tracy M Jones, Jessica D Shaw, Joseph S Staffetti, Tetsuo Ashizawa, Sheng-Han Kuo, Brent L Fogel, George R Wilmot, Susan L Perlman, Chiadi U Onyike and 2 more

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Neurodegenerative disease management, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 34 papers.

0numbers the graph read from it
0cells of the map it votes in
34citing papers in PubMed
6.0field-weighted citation impact, top 3% of its field
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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

34 citing papers in PubMed, 62 citations in OpenAlex.

  1. Trial
  2. Article
  3. Article
  4. Review
  5. Article
  6. Article
  7. Review
  8. Article
  9. Article
  10. Article
  11. Article
  12. Article
  13. Article
  14. Article
  15. Observational
  16. Molecular Mechanisms and Therapeutics for the GAA·TTC Expansion Disease Friedreich Ataxia.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2019
    Review
  17. Measuring peripheral nerve involvement in Friedreich's ataxia.Annals of clinical and translational neurology · 2019
    Article
  18. Ataxia.Continuum (Minneapolis, Minn.) · 2019
    Review
  19. Review
  20. Phosphodiesterase Inhibitors Revert Axonal Dystrophy in Friedreich's Ataxia Mouse Model.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2019
    Article
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

12 authors at 8 institutions in 1 country.

Tanya V ArancaUniversity of South Florida Ataxia Research Center, Department of Neurology, FL, USA.
Tracy M JonesUniversity of South Florida Ataxia Research Center, Department of Neurology, FL, USA.
Jessica D ShawUniversity of South Florida Ataxia Research Center, Department of Neurology, FL, USA.
Joseph S StaffettiUniversity of South Florida Ataxia Research Center, Department of Neurology, FL, USA.
Tetsuo AshizawaMcKnight Brain Institute, University of Florida Department of Neurology, FL, USA.
Sheng-Han KuoDepartment of Neurology, Columbia University, NY, USA.
Brent L FogelDepartment of Neurology, Neurogenetics Program, David Geffen School of Medicine, University of California, CA, USA.
George R WilmotDepartment of Neurology, Emory University, GA, USA.
Susan L PerlmanAtaxia and Huntington Disease Center of Excellence, University of California, CA, US.
Chiadi U OnyikeDepartment of Psychiatry and Behavioral Sciences, Johns Hopkins University school of Medicine MD, USA.
Sarah H YingDepartment of Neurology, Johns Hopkins University School of Medicine, MD, USA.
Theresa A ZesiewiczUniversity of South Florida Ataxia Research Center, Department of Neurology, FL, USA.
University of South Florida · USAllen Institute for Brain Science · USColumbia University · USEmory University · USHuntington University · USJohns Hopkins Medicine · USJohns Hopkins University · USUniversity of California System · US

Funding

Cerebellar Circuitry in the Pathophysiology of TremorR01NS104423 · NINDS · COLUMBIA UNIVERSITY HEALTH SCIENCES · PI KUO, SHENG-HAN · 2018 to 2022
$2.0M
Rare and Novel Genetic Variation in the Pathogenesis of Cerebellar AtaxiaR01NS082094 · NINDS · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI FOGEL, BRENT LINDEN · 2013 to 2017
$1.7M
The role of glucocerebrosidase and chaperone_mediated autophagy in Parkinsons disK08NS083738 · NINDS · COLUMBIA UNIVERSITY HEALTH SCIENCES · PI KUO, SHENG-HAN · 2013 to 2017
$940k
NINDS NIH HHS K08 NS083738NINDS NIH HHS R01 NS082094NINDS NIH HHS R01 NS104423
6 · The paper itself

Abstract

Friedreich's ataxia (FRDA) is an inherited, progressive neurodegenerative disease that typically affects teenagers and young adults. Therapeutic strategies and disease insight have expanded rapidly over recent years, leading to hope for the FRDA population. There is currently no US FDA-approved treatment for FRDA, but advances in research of its pathogenesis have led to clinical trials of potential treatments. This article reviews emerging therapies and discusses future perspectives, including the need for more precise measures for detecting changes in neurologic symptoms as well as a disease-modifying agent.

Indexed as

AdolescentClinical Trials as TopicDrug Therapy, CombinationFriedreich AtaxiaGenetic TherapyHumansNerve Growth FactorsSeverity of Illness IndexTreatment OutcomeNerve Growth FactorsantioxidantserythropoietinFRDAFriedreich's ataxiaHDACiron

Identifiers

PMID26782317
PMCPMC4768799
OpenAlexW3163189904

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

None linked

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.