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Record W4401363365 · doi:10.3233/trd-240069

Rett syndrome: A coming of age

2024· article· en· W4401363365 on OpenAlexaboutno aff
Alan K. Percy

Bibliographic record

VenueTranslational Science of Rare Diseases · 2024
Typearticle
Languageen
FieldBiochemistry, Genetics and Molecular Biology
TopicGenetics and Neurodevelopmental Disorders
Canadian institutionsnot available
FundersNational Institutes of Health
KeywordsRett syndromeNatural historyNatural history studyMedicineMECP2EtiologyClinical trialNeurologyPsychiatryPediatricsPsychologyNeurosciencePathologyBiologyInternal medicineGenetics

Abstract

fetched live from OpenAlex

Rett syndrome (RTT) was first recognized in the late 1950s by Andreas Rett in Vienna and Bengt Hagberg in Uppsala. Hagberg, following a meeting with Rett, decided to call the disorder Rett syndrome in the landmark paper which appeared in the Annals of Neurology in 1983. That report led to the worldwide recognition of this relatively young and unique neurodevelopmental disorder, the concerted effort to establish its epidemiology, etiology, and natural history, and the establishment of clinical criteria for its diagnosis. Our understanding of RTT progressed rapidly, in part due to the remarkable diagnostic advances in genetics linking RTT with variations in the methyl-CpG-binding protein 2 (MECP2) gene at Xq28. In 2003, the NIH funded a Natural History study of RTT and related disorders which provided critical cross-sectional and longitudinal data that resulted in the increased understanding of RTT, the development of better management strategies, and an increase in pharmaceutical and gene-based products designed to provide specific therapies. The FDA-approved oral agent trofinetide has been shown to provide incremental improvements in the core features of RTT. Two gene-based therapies are currently being assessed in clinical trials in Canada and the US. Additional treatment strategies are being assessed at the clinical and translational levels.

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)0.000
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 score0.815
Threshold uncertainty score0.198

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 head0.010
GPT teacher head0.256
Teacher spread0.246 · 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 teacher head, not a consensus.

The models applied no category: nothing in the taxonomy fit this work.
Study designBench or experimental
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
Published2024
Admission routes1
Has abstractyes

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