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Record W4417458373 · doi:10.1212/nxg.0000000000200334

<i>DEGS1</i> -Related Hypomyelinating Leukodystrophy

2025· article· en· W4417458373 on OpenAlex
M Grinberg, Breanne Dale, Rajesh RamachandranNair, Mark A. Tarnopolsky, Robyn Whitney, Resham Ejaz

Why this work is in the frame

A frame that forgets how it found something cannot be audited. These are the routes that admitted this work.

affAt least one author lists a Canadian institution in the pinned OpenAlex snapshot.

Bibliographic record

VenueNeurology Genetics · 2025
Typearticle
Languageen
FieldBiochemistry, Genetics and Molecular Biology
TopicRNA regulation and disease
Canadian institutionsMcMaster Children's HospitalMcMaster UniversityMcMaster University Medical Centre
Fundersnot available
KeywordsLeukodystrophyNatural historyPhenotypeStroke (engine)Clinical phenotype

Abstract

fetched live from OpenAlex

Objectives: -related leukodystrophy with phenotypic heterogeneity both within the family and in previously reported individuals. Methods: Four related individuals were followed by the neurology, genetics, and neurometabolic teams with clinical examinations, MRI brain, and genetic/metabolic investigations. Trio exome sequencing (ES) was completed after initial investigations returned normal. Results: , c.110T>C (p.Met37Thr), for the proband with subsequent confirmatory testing of remaining affected family members. While the affected individuals share many similarities, there is notable variability in severity between them as well as with other 33 previously reported individuals, including 2 with the same pathogenic variant. This variability is highlighted in the presence of seizures, nystagmus, failure to thrive, microcephaly, MRI brain findings, and age at disease onset. Discussion: -related leukodystrophy and adds to the current understanding of the phenotypic range of affected individuals. Consideration of variability in the condition's natural history and outcome is key to appropriately counsel patients and families.

Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.

Full frame distilled prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.

metaresearch head score (Codex)0.000
metaresearch head score (Gemma)0.000
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesnone
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Bench or experimental · Consensus signal: none
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.691
Threshold uncertainty score0.586

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0000.000
Meta-epidemiology (narrow)0.0000.000
Meta-epidemiology (broad)0.0000.000
Bibliometrics0.0000.000
Science and technology studies0.0000.000
Scholarly communication0.0000.000
Open science0.0000.000
Research integrity0.0000.000
Insufficient payload (model declined to judge)0.0000.000

Machine scores (provisional)

The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.

Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.

Opus teacher head0.005
GPT teacher head0.236
Teacher spread0.231 · how far apart the two teachers sit on this one work
Validation statusscore_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from it