Evidence map›Paper›PMID 23859349›Full record

ReviewJournal of neurochemistry2013

Deferiprone for the treatment of Friedreich's ataxia.

Massimo Pandolfo, Laura Hausmann

Registry-linked trialAbstract readReview
PubMed Publisher
In one paragraph

Review in Journal of neurochemistry, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02882477 (Treatment of Wolfram Syndrome Type 2 With the Chelator Deferiprone, and Incretin Based Therapy), which is not on this map. Cited by 42 papers.

0numbers the graph read from it
0cells of the map it votes in
42citing papers in PubMed
4.2field-weighted citation impact, top 5% 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.

NCT02882477 phase2 / phase3unknown statusstarted 2016, after this paper: background citation

Treatment of Wolfram Syndrome Type 2 With the Chelator Deferiprone, and Incretin Based Therapy

Ran2016Enrolled20Registered outcomes6Posted comparisons0ConditionsDiabetes Mellitus, Gastroduodenal Ulcer, Iron Metabolism Disorders, Optic AtrophyArmsacetylcysteine, Deferiprone, Sitagliptin and Metformin
Open the trial in the graph
3 · Its place in the literature

Who cites it

42 citing papers in PubMed, 84 citations in OpenAlex.

  1. Article
  2. Article
  3. Review
  4. Metal Ion Signaling in Biomedicine.Chemical reviews · 2025
    Review
  5. Review
  6. Article
  7. Review
  8. Iron imbalance in neurodegeneration.Molecular psychiatry · 2024
    Review
  9. Review
  10. Mitochondrial Inherited Disorders and their Correlation with Neurodegenerative Diseases.Endocrine, metabolic & immune disorders drug targets · 2024
    Review
  11. Iron Dyshomeostasis and Mitochondrial Function in the Failing Heart: A Review of the Literature.American journal of cardiovascular drugs : drugs, devices, and other interventions · 2024
    Review
  12. Review
  13. Article
  14. Article
  15. Article
  16. Review
  17. Emerging therapies in Friedreich's Ataxia.Expert review of neurotherapeutics · 2020
    Review
  18. Review
  19. Review
  20. Review
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

2 authors at 2 institutions in 2 countries.

Massimo PandolfoDepartment of Neurology, Université Libre de Bruxelles, Hôpital Erasme, Brussels, Belgium. massimo.pandolfo@ulb.ac.be
Laura Hausmann
Erasmus Hospital · BEUniversitätsklinikum Aachen · DE

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Friedreich's ataxia (FRDA) is a neurological disease related to a deficiency of the protein frataxin involved in iron-sulfur (Fe-S) cluster biogenesis. This leads to an increased cellular iron uptake accumulating in mitochondria, and a subsequently disturbed iron homeostasis. The detailed mechanism of iron regulation of frataxin expression is yet unknown. Deferiprone, an iron chelator that may cross the blood-brain barrier, was shown to shuttle iron between subcellular compartments. It could also transfer iron from iron-overloaded cells to extracellular apotransferrin and pre-erythroid cells for heme synthesis. Here, clinical studies on Deferiprone are reviewed in the context of alternative agents such as desferoxamine, with specific regard to its mechanistic and clinical implications.

Indexed as

AnimalsClinical Trials as TopicDeferiproneDrug Evaluation, PreclinicalFrataxinFriedreich AtaxiaHumansIronIron-Binding ProteinsIron Chelating AgentsPyridonesSulfurDeferiproneFrataxinIronIron-Binding ProteinsIron Chelating AgentsPyridonesSulfurclinical studyDeferipronefrataxinFriedreich's ataxia (FRDA)

Identifiers

PMID23859349
OpenAlexW2058333232

What OpenQuestion holds

Textmetadata
Read underepoch 390

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.