Evidence map›Paper›PMID 41743237›Full record

ArticleiScience2026

Sphingolipid-neutralizing molecular therapy reduces psychosine cytotoxicity in Krabbe disease.

Salma Begum, Shin-Chang Hsueh, Ezra M Y Cheria, Jayar Espejo, Ping Zhang, Armand Collin, Edgar Kappauf, Murielle Mardenli, Michael H Gelb, Chang-Chun Ling and 1 more

Abstract read
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In one paragraph

Article in iScience, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

11 authors.

Salma BegumDepartment of Pediatrics (Genetics and Metabolism), Columbia University Irving Medical Center, New York, NY 10032, USA.
Shin-Chang HsuehDepartment of Pediatrics (Genetics and Metabolism), Columbia University Irving Medical Center, New York, NY 10032, USA.
Ezra M Y CheriaBiomarkers Core Laboratory (BCL) at the Irving Institute for Clinical and Translational Research, Columbia University Irving Medical Center, New York, NY 10032, USA.
Jayar EspejoDepartment of Chemistry, University of Calgary, Calgary, AB T2N 1N4, Canada.
Ping ZhangDepartment of Chemistry, University of Calgary, Calgary, AB T2N 1N4, Canada.
Armand CollinNeuroPoly Lab, Institute of Biomedical Engineering, Polytechnique Montréal, Université de Montréal, Montréal, QC H3T 1J4, Canada.
Edgar KappaufNeuroPoly Lab, Institute of Biomedical Engineering, Polytechnique Montréal, Université de Montréal, Montréal, QC H3T 1J4, Canada.
Murielle MardenliNeuroPoly Lab, Institute of Biomedical Engineering, Polytechnique Montréal, Université de Montréal, Montréal, QC H3T 1J4, Canada.
Michael H GelbDepartment of Chemistry, University of Washington, Seattle, WA 98195, USA.
Chang-Chun LingDepartment of Chemistry, University of Calgary, Calgary, AB T2N 1N4, Canada.
Gustavo H B MaegawaDepartment of Pediatrics (Genetics and Metabolism), Columbia University Irving Medical Center, New York, NY 10032, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The deficiency of β-galactosylceramidase causes a lysosomal leukodystrophy, known as Krabbe disease (KD), resulting in elevated psychosine (PSY) levels, which are highly cytotoxic to myelin-forming cells. 2-hydroxypropyl-α-CD (HPaCD), a cyclic-oligosaccharide containing a lipophilic central cavity and hydrophilic outer surfaces, significantly reduces PSY cytotoxicity in cultured KD patient cells. Further

Indexed as

MedicineNeurologyPathophysiology

Identifiers

PMID41743237
PMCPMC12930047

What OpenQuestion holds

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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.