Evidence map›Paper›PMID 31686946›Full record

ArticleDegenerative neurological and neuromuscular disease2019

Orphan Drugs In Development For The Treatment Of Friedreich's Ataxia: Focus On Omaveloxolone.

Shaila D Ghanekar, Wai Wai Miller, Colin J Meyer, Kevin J Fenelon, Alvin Lacdao, Theresa A Zesiewicz

Open access · diamondAbstract read
In one paragraph

Article in Degenerative neurological and neuromuscular disease, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed
–field-weighted citation impact, top 90% 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

6 citing papers in PubMed, 8 citations in OpenAlex.

  1. Review
  2. Development of an AAV-based gene therapy for the ocular phenotype of Friedreich's ataxia.Molecular therapy : the journal of the American Society of Gene Therapy · 2026
    Article
  3. Article
  4. Article
  5. Review
  6. 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

6 authors at 2 institutions in 2 countries.

Shaila D GhanekarUniversity of South Florida (USF), Department of Neurology, Tampa, FL, USA.
Wai Wai MillerUniversity of South Florida (USF), Department of Neurology, Tampa, FL, USA.ORCID 0000-0003-2913-6676
Colin J MeyerReata Pharmaceuticals, Inc., Department of Product Development, Irving, TX, USA.
Kevin J FenelonUniversity of South Florida (USF), Department of Neurology, Tampa, FL, USA.
Alvin LacdaoUniversity of South Florida (USF), Department of Neurology, Tampa, FL, USA.
Theresa A ZesiewiczUniversity of South Florida (USF), Department of Neurology, Tampa, FL, USA.
University of South Florida · USReata Pharmaceuticals (United States) · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Friedreich's Ataxia (FRDA) is a devastating and progressive ataxia, marked by mitochondrial dysfunction and oxidative stress. Nrf2 activators such as omaveloxolone (Omav) modulate antioxidative mechanisms, and thus may be viable therapeutic agents in FRDA.

Indexed as

FRDANrf2 activatorsOmavomaveloxoloneoxidative stress

Identifiers

PMID31686946
PMCPMC6800542
OpenAlexW2980119243

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
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.