Evidence map›Paper›PMID 41560433›Full record

ReviewAnnals of clinical and translational neurology2026

Clinically Relevant Outcome Measures in Women With Adrenoleukodystrophy.

Chenwei Yan, Elizabeth I Pierpont, Amena S Fine, Reena V Kartha

Abstract readReview
In one paragraph

Review in Annals of clinical and translational neurology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. Nervonic acid supplementation mitigates disease severity biomarkers in adrenoleukodystrophy.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2026
    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

4 authors.

Chenwei YanDepartment of Experimental and Clinical Pharmacology, College of Pharmacy, Center for Orphan Drug Research, University of Minnesota Twin Cities, Minneapolis, Minnesota, USA.
Elizabeth I PierpontDivision of Clinical Behavioral Neuroscience, Medical School, University of Minnesota Twin Cities, Minneapolis, Minnesota, USA.ORCID 0000-0001-7555-8613
Amena S FineDepartment of Neurogenetics, Moser Center for Leukodystrophies, Kennedy Krieger Institute, Baltimore, Maryland, USA.ORCID 0000-0002-6855-8807
Reena V KarthaDepartment of Experimental and Clinical Pharmacology, College of Pharmacy, Center for Orphan Drug Research, University of Minnesota Twin Cities, Minneapolis, Minnesota, USA.ORCID 0000-0002-1692-1539

Funding

University of Minnesota Clinical and Translational Science Institute (UMN CTSI)UM1TR004405 · NCATS · UNIVERSITY OF MINNESOTA · PI Bruce R Blazar, Damien A Fair · 2023 to 2026
$30.8M
Neurocognitive and neuroimaging markers of emerging cerebral adrenoleukodystrophyK23NS123258 · NINDS · UNIVERSITY OF MINNESOTA · PI Elizabeth Irene Pierpont · 2022 to 2026
$794k
National Institute for Neurological Disorders and Stroke K23NS123258NCATS NIH HHS UM1TR004405NINDS NIH HHS K23 NS123258
6 · The paper itself

Abstract

Adrenoleukodystrophy is a rare inherited peroxisomal disease caused by pathogenic variants in the ABCD1 gene located on the X chromosome. Although the most severe central nervous system and adrenal complications typically affect only men with adrenoleukodystrophy, the majority of women develop myeloneuropathy symptoms in adulthood. In observational studies of women with adrenoleukodystrophy, several clinical rating scales have been used to assess disease manifestations and capture differences between asymptomatic and symptomatic women. To facilitate development of treatments to address symptoms in women, there is a need to identify clinical outcome measures that can sensitively assess disease progression and treatment responses. The goals of this scoping review were to: (1) identify and review clinically relevant assessment scales that have been utilized to capture disease manifestations in women with adrenoleukodystrophy, and (2) provide recommendations on key objectives for further research.

Indexed as

AdrenoleukodystrophyOutcome Assessment, Health CareFemaleHumansadrenoleukodystrophyclinical outcomesmyeloneuropathywomen

Identifiers

PMID41560433
PMCPMC13071096

What OpenQuestion holds

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Registered trials

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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.