Evidence map›Paper›PMID 42260209›Full record

ArticleNeurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology2026

Intracranial calcifications in DEGS1-Related Leukodystrophy: a potentially under-recognised neuroimaging feature.

Ylenia Vaia, Neena Kim, Sharmila Jeyasingh, Sniya Sudhakar, Kshitij Mankad, Asthik Biswas, Ata Siddiqui, Helen Mundy, Tammy Hedderly, Rahul R Singh

Abstract readCase Reports
In one paragraph

Article in Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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0citing papers in PubMed
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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

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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

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5 · Who and what money

Authors and funding

10 authors.

Ylenia VaiaDepartment of Biomedical and Clinical Sciences, Neuroscience Research Centre, University of Milan, Milan, Italy.
Neena KimDepartment of Neurology, Great Ormond Street Hospital for Children, London, UK.
Sharmila JeyasinghDepartment of Paediatrics, Royal Alexandra Children's Hospital, Brighton, UK.
Sniya SudhakarDepartment of Radiology, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.
Kshitij MankadDepartment of Radiology, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.
Asthik BiswasDepartment of Radiology, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.
Ata SiddiquiDepartment of Neuroradiology, Evelina London Children's Hospital at Guys and St Thomas' NHS Foundation Trust, London, UK.
Helen MundyDepartment of Inherited Metabolic Disease, Evelina London Children's Hospital, London, UK.
Tammy HedderlyChildren's Neurosciences, Evelina London Children's Hospital at Guys and St Thomas' NHS Foundation Trust, Wstminster Bridge Road, London, SE1 7EH, UK.
Rahul R SinghChildren's Neurosciences, Evelina London Children's Hospital at Guys and St Thomas' NHS Foundation Trust, Wstminster Bridge Road, London, SE1 7EH, UK. rahul.singh7@nhs.net.ORCID http://orcid.org/0000-0001-9689-8393

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Pathogenic variants in DEGS1, encoding a sphingolipid desaturase critical for ceramide biosynthesis, disrupt sphingolipid homeostasis and oligodendrocyte function, leading to abnormal myelination. We report an infant with genetically confirmed DEGS1-related leukodystrophy (homozygous c.337A > C, p.Asn113His) who presented with abnormal eye movements and early-onset developmental arrest. This was accompanied by axial hypotonia, severe feeding difficulties, and refractory epilepsy, including epileptic spasms with modified hypsarrhythmia. Brain MRI demonstrated diffuse abnormal myelination per age, thin corpus callosum, and cerebellar involvement. Notably, susceptibility-weighted imaging suggested cerebellar white matter calcifications, which were confirmed on brain CT, alongside punctate supratentorial calcifications, an atypical finding for DEGS1-related disease. This case expands the neuroimaging phenotype of DEGS1-related leukodystrophy and highlights intracranial calcifications, particularly within the cerebellum, as a potential diagnostic clue in the differential diagnosis of hypomyelinating disorders with conatal onset. Our findings also underscore the severe clinical course associated with DEGS1 deficiency, including profound developmental impairment, early-onset epilepsy, and persistent feeding difficulties.

Indexed as

CalcinosisHereditary Central Nervous System Demyelinating DiseasesBrainHumansInfantMagnetic Resonance ImagingMaleNeuroimaginghypomyelinating leukodystrophyIntracranial calcificationSphingolipid biosynthesis

Identifiers

PMID42260209
PMCPMC13246557

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