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Record W4206047998 · doi:10.1017/cjn.2021.334

P.053 A clinical mystery finally revealed

2021· article· en· W4206047998 on OpenAlexvenueno aff
Z Aljohani, S Venance

Bibliographic record

VenueCanadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques · 2021
Typearticle
Languageen
FieldBiochemistry, Genetics and Molecular Biology
TopicNuclear Structure and Function
Canadian institutionsnot available
Fundersnot available
KeywordsMyopathyMuscular dystrophyMedicineExonAtrophyPhenotypeMutationTrunkInternal medicinePathologyCardiologyGeneticsBiologyGene

Abstract

fetched live from OpenAlex

Background: We report 2 brothers sharing FHL1 identified mutation. They presented in childhood with overlapping clinical features characterized by early onset stiffness and increased muscle definition with cardiac involvement. After 30 years of neurological followup, the diagnosis is finally revealed. Methods: At early ages, both had increased definition of upper trunk musculature. The older brother had hypophonic voice with raspy character, which is to our knowledge, not reported with this mutation before. He required a pacemaker for arrhythmias, while the younger developed congestive heart failure. Results: Their initial investigations failed to unveil a diagnosis, including a negative next generation sequencing (NGS) panel for AR LGMD. An expanded NGS sent on the older brother revealed he is hemizygous for 1770 bp deletion within FHL 1 gene, this deletion includes exon 7to 8, and confirmed on the other. Conclusions: First reported in 2008, FHL1 mutations result in phenotypically distinct neuromuscular disorders: X-linked myopathy with Postural Muscle Atrophy and generalized hypertrophy, X-linked dominant scapuloperoneal Myopathy, and Reducing Body Myopathy. Subsequently other phenotypes have been reported including Emery-Dreifuss muscular dystrophy and hypertrophic cardiomyopathy. Our patients present with a phenotype that had been reported with FHL1 mutation, highlighting the possible recognition of this presentation in aiding a diagnostic approach.

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.

How this classification was reachedexpand

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)0.003
metaresearch head score (Gemma)0.003
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesScience and technology studies
Consensus categoriesScience and technology studies
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Observational · Consensus signal: Observational
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.374
Threshold uncertainty score1.000

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0030.003
Meta-epidemiology (narrow)0.0000.000
Meta-epidemiology (broad)0.0000.000
Bibliometrics0.0000.001
Science and technology studies0.0010.003
Scholarly communication0.0000.000
Open science0.0010.000
Research integrity0.0000.001
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 head0.034
GPT teacher head0.286
Teacher spread0.252 · 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

Classification

machine, unvalidated

Machine predicted; both teacher heads agree on what is shown here.

Study designObservational
Domainnot available
GenreEmpirical

How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".

Quick stats

Citations0
Published2021
Admission routes1
Has abstractyes

Explore more

Same venueCanadian Journal of Neurological Sciences / Journal Canadien des Sciences NeurologiquesSame topicNuclear Structure and FunctionFrench-language works237,207