Evidence map›Paper›PMID 37746147›Full record

ArticleFrontiers in neuroscience2023

Rationale and protocol of a double-blind, randomized, placebo-controlled trial to test the efficacy, safety, and tolerability of dimethyl fumarate in Friedreich Ataxia (DMF-FA-201).

Chiara Pane, Alberto Maria Marra, Ludovica Aliberti, Mario Campanile, Federica Coscetta, Giulia Crisci, Roberta D'Assante, Angela Marsili, Giorgia Puorro, Andrea Salzano and 2 more

Abstract read
In one paragraph

Article in Frontiers in neuroscience, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
–field-weighted citation impact
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

8 citing papers in PubMed.

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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.

Chiara PaneDepartment of Neurosciences and Reproductive and Odontostomatological Sciences, Federico II University, Naples, Italy.
Alberto Maria MarraDepartment of Translational Medical Sciences, Federico II University, Naples, Italy.
Ludovica AlibertiDepartment of Translational Medical Sciences, Federico II University, Naples, Italy.
Mario CampanileDepartment of Neurosciences and Reproductive and Odontostomatological Sciences, Federico II University, Naples, Italy.
Federica CoscettaDepartment of Translational Medical Sciences, Federico II University, Naples, Italy.
Giulia CrisciDepartment of Translational Medical Sciences, Federico II University, Naples, Italy.
Roberta D'AssanteDepartment of Translational Medical Sciences, Federico II University, Naples, Italy.
Angela MarsiliDepartment of Neurosciences and Reproductive and Odontostomatological Sciences, Federico II University, Naples, Italy.
Giorgia PuorroDepartment of Neurosciences and Reproductive and Odontostomatological Sciences, Federico II University, Naples, Italy.
Andrea SalzanoCardiac Unit, AORN A Cardarelli, Naples, Italy.
Antonio CittadiniDepartment of Translational Medical Sciences, Federico II University, Naples, Italy.
Francesco SaccàDepartment of Neurosciences and Reproductive and Odontostomatological Sciences, Federico II University, Naples, Italy.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Introduction: Friedreich Ataxia (FRDA) is an autosomal recessive neurodegenerative disorder that causes gait and limb ataxia, dysarthria, and impaired vibratory sense, with cardiomyopathy being the predominant cause of death. There is no approved therapy, which results in the use of symptomatic treatments and the chronic support of physiotherapy. Dimethyl fumarate (DMF) is a fumaric acid ester used for the treatment of psoriasis and Multiple Sclerosis (MS). It induces Nrf2 Methods: The aim of our study is to investigate if DMF can increase the expression of the Endpoints: The primary endpoint will be a change in Conclusions: This is the first study aimed at exploring the ability of DMF, an already available treatment for MS and psoriasis, to correct the biological deficits of FRDA and potentially improve mitochondrial respiration

Indexed as

clinical outcomesclinical study designdimethyl fumaratefrataxin levelFriedreich Ataxiamitochondrial biogenesisNrf2 pathwaysafety

Identifiers

PMID37746147
PMCPMC10513368

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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.