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Record W2143679314 · doi:10.1136/jmg.38.11.e40

Molecular and clinical study of two myotonic dystrophy homozygotes

2001· letter· en· W2143679314 on OpenAlexaboutno aff
Fahri Akbaş

Bibliographic record

VenueJournal of Medical Genetics · 2001
Typeletter
Languageen
FieldNeuroscience
TopicGenetic Neurodegenerative Diseases
Canadian institutionsnot available
Fundersnot available
KeywordsMyotonic dystrophyGeneticsMedicineMyotoniaBioinformaticsComputational biologyBiology

Abstract

fetched live from OpenAlex

Editor—Myotonic dystrophy (DM) is the most common form of inherited neuromuscular disease in adults and is characterised by progressive muscle wasting and myotonia. The mutation responsible for DM has been identified as the amplification of a polymorphic (CTG)n repeat in the 3′ untranslated region of a gene encoding a serine/threonine kinase ( DMPK ).1-2 The DM trinucleotide repeat is highly polymorphic in normal subjects, ranging from 5 to 37 CTG repeats, while 1000 or more CTG repeats have been observed in congenital DM cases.1-3 It is now generally accepted that the CTG repeat length is correlated with clinical severity and the age at onset of the disease; therefore, genetic tests are essential in the monitoring and management of DM patients and their family members.4-6 Homozygous DM cases have very rarely been published.7 8It was assumed that the homozygous state was lethal in DM or at least so severe that it would lead to intrauterine death. Cobo et al 7 studied a consanguineous French-Canadian family in which two sisters were homozygous for the “at risk” haplotype but were asymptomatic and showed no evidence of DM on extensive clinical examination. Both sisters possessed two alleles with repeat sizes normally seen in minimally affected patients. Both parents were affected. Martorell et al 8 described three unrelated homozygous myotonic dystrophy patients. One patient had the classical form of myotonic dystrophy and the other two were mildly affected. A remarkable feature was the mildness of the phenotype in the homozygous patients; one, for example, had late onset cataract as the only manifestation. Along with the observations of Cobo et al 7and Martorell …

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 machine prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.

metaresearch head score (Codex)0.001
metaresearch head score (Gemma)0.002
Version: metacan-v3-hybrid-931329e0061cValidation status: machine_predicted_unvalidated
Candidate categoriesnone
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Case report · Consensus signal: Case report
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.008
Threshold uncertainty score0.025

Distilled classifier scores by category (both heads)

CategoryCodexGemma
Metaresearch0.0010.002
Meta-epidemiology (narrow)0.0010.000
Meta-epidemiology (broad)0.0010.001
Bibliometrics0.0010.000
Science and technology studies0.0010.001
Scholarly communication0.0010.001
Open science0.0010.001
Research integrity0.0020.001
Insufficient payload (model declined to judge)0.0080.002

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 head0.054
GPT teacher head0.371
Teacher spread0.317 · 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; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.

The models applied no category: nothing in the taxonomy fit this work.
Study designCase report
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

Citations5
Published2001
Admission routes1
Has abstractyes

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