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Record W4408694309 · doi:10.1016/j.gimo.2025.103425

Autosomal dominant HK1-related neurodevelopmental disorder with visual defects and brain anomalies (NEDVIBA): An emerging mitochondrial disorder

2025· article· en· W4408694309 on OpenAlex
Bobby G. Ng, Erik A. Eklund, Jill A. Rosenfeld, Abdallah F. Elias, Aya Abu‐El‐Haija, Céline Bris, Magalie Barth, Jong‐Hee Chae, Murim Choi, Holly Dubbs, Carl Fratter, Nicola Foulds, Candace Gamble, Ralitza H. Gavrilova, Jaclyn Haven, Trevor L. Hoffman, Jill V. Hunter, Austin Larson, Timothy E. Lotze, Pilar Magoulas, Emily Magness, Debra M Bootin, Eric D. Marsh, Victoria Nesbitt, Matthew Pastore, Joanna Poulton, Shamima Rahman, Fernando Scaglia, Chaya N. Murali, Jennifer E. Posey, Joshua Rotenberg, Betsy Schmalz, Deepali N. Shinde, Zöe Powis, Rivka Sukenik‐Halevy, Kristen Truxal, Tami Uster, Matheus Vernet Machado Bressan Wilke, Eric W. Klee, H. Woo, Donald Younkin, Jorge L. Granadillo, Seema R. Lalani, David Chitayat, Wendy K. Chung, Hudson H. Freeze, Volkan Okur

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.
fundA Canadian funder is recorded on the work.

Bibliographic record

VenueGenetics in Medicine Open · 2025
Typearticle
Languageen
FieldBiochemistry, Genetics and Molecular Biology
TopicMitochondrial Function and Pathology
Canadian institutionsHospital for Sick Children
FundersUniversity of Colorado School of Medicine, Anschutz Medical CampusEunice Kennedy Shriver National Institute of Child Health and Human DevelopmentTexas Children's HospitalNational Human Genome Research InstituteHospital for Sick ChildrenGreat Ormond Street Institute of Child HealthOxford University Hospitals NHS Foundation TrustGreat Ormond Street Hospital for ChildrenTel Aviv UniversitySeoul National UniversityNational Institute of Child Health and Human DevelopmentNational Institute of Diabetes and Digestive and Kidney DiseasesChildren's Hospital of PhiladelphiaLunds UniversitetChungbuk National UniversityQuest DiagnosticsCentre National de la Recherche ScientifiqueNIHR Great Ormond Street Hospital Biomedical Research CentreSeoul National University HospitalNationwide Children's HospitalUniversity Hospital Southampton NHS Foundation TrustUniversity of OxfordCollege of Medicine, Seoul National UniversityInstitut National de la Santé et de la Recherche MédicaleKaiser Permanente
KeywordsNeurodevelopmental disorderNeurosciencePsychologyMedicineDevelopmental psychology

Abstract

fetched live from OpenAlex

Purpose: ) encodes a ubiquitously expressed hexokinase, which is responsible for the first step of glycolysis, phosphorylation of glucose to glucose-6-phosphate. Both autosomal recessive and dominant variants in this gene have previously been shown to cause human disease, and presently, there are clinical data available for 27 individuals with the monoallelic neurodevelopmental disorder with visual defects and brain anomalies. Delineation of the entire phenotypic spectrum and genotype-phenotype relations will aid in management and counseling decisions. Methods: . We also reviewed data from the published literature. Results: The clinical manifestations of neurodevelopmental disorder with visual defects and brain anomalies include varying degrees of intellectual disability/developmental delay, hypotonia, epileptic encephalopathy, visual deficits, a Leigh syndrome spectrum pattern on brain magnetic resonance imaging, and elevated lactate in blood and cerebrospinal fluid, suggesting mitochondrial dysfunction. Based on severity, individuals can be classified into mild, moderate, severe, or lethal forms. In terms of genotype-phenotype correlation, we find that all individuals carrying a missense variant at the threonine 457 residue have severe clinical features. Conclusion: should be included in mitochondrial disorder gene sequencing panels.

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 score0.724
Threshold uncertainty score0.904

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 head0.009
GPT teacher head0.300
Teacher spread0.291 · 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