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Record W4391112194 · doi:10.1002/mgg3.2363

Lessons from two series by physicians and caregivers' self‐reported data in <i>DDX3X</i>‐related disorders

2024· article· en· W4391112194 on OpenAlexafffund
Valentin Ruault, Pauline Burger, Johanna Gradels‐Hauguel, Nathalie Soriano Ruiz, Rami Abou Jamra, Alexandra Afenjar, Yves Alembik, Jean‐Luc Alessandri, Stéphanie Arpin, Giulia Barcia, Šárka Bendová, Ange‐Line Bruel, Perrine Charles, Nicolas Chatron, Maya Chopra, Solène Conrad, Valérie Cormier‐Daire, Auriane Cospain, Christine Coubes, Juliette Coursimault, Andrée Delahaye‐Duriez, Martine Doco, William Dufour, Benjamin Durand, Camille Engel, Laurence Faivre, Fanny Ferroul, Mélanie Fradin, Hélène Frenkiel, Carlo Fusco, Livia Garavelli, Aurore Garde, Bénédicte Gérard, David Germanaud, Louise Goujon, Aurélie Gouronc, Emmanuelle Ginglinger, Alice Goldenberg, Miroslava Hančárová, Markéta Havlovičová, Delphine Héron, Bertrand Isidor, Nolwenn Jean Marçais, Boris Keren, Margarete Koch‐Hogrebe, Paul Kuentz, Victoria Lamure, Anne-Sophie Lèbre, François Lecoquierre, Natacha Lehman, Gaëtan Lesca, Stanislas Lyonnet, Delphine Martin, Cyril Mignot, Teresa Neuhann, Gaël Nicolas, Mathilde Nizon, Florence Petit, Christophe Philippe, Amélie Piton, Marzia Pollazzon, Darina Prchalová, Audrey Putoux, Marlène Rio, Sophie Rondeau, Massimiliano Rossi, Quentin Sabbagh, Pascale Saugier-Véber, Ariane Schmetz, Julie Steffann, Christel Thauvin‐Robinet, Annick Toutain, Frédéric Tran Mau‐Them, Gabriele Trimarchi, Marie Vincent, Markéta Vlčková, Dagmar Wieczorek, Marjolaine Willems, Kévin Yauy, Michaela Zelinová, Alban Ziegler, Boris Chaumette, Bekim Sadiković, Jean‐Louis Mandel, David Geneviève

Bibliographic record

VenueMolecular Genetics & Genomic Medicine · 2024
Typearticle
Languageen
FieldBiochemistry, Genetics and Molecular Biology
TopicGenetics and Neurodevelopmental Disorders
Canadian institutionsLondon Health Sciences CentreWestern UniversityMcGill University
FundersFondation Bettencourt SchuellerEunice Kennedy Shriver National Institute of Child Health and Human DevelopmentMinistère des Affaires Sociales et de la SantéGenome Canada
KeywordsSeries (stratigraphy)MedicineData scienceBiologyComputer science

Abstract

fetched live from OpenAlex

INTRODUCTION AND METHODS: We report two series of individuals with DDX3X variations, one (48 individuals) from physicians and one (44 individuals) from caregivers. RESULTS: These two series include several symptoms in common, with fairly similar distribution, which suggests that caregivers' data are close to physicians' data. For example, both series identified early childhood symptoms that were not previously described: feeding difficulties, mean walking age, and age at first words. DISCUSSION: Each of the two datasets provides complementary knowledge. We confirmed that symptoms are similar to those in the literature and provides more details on feeding difficulties. Caregivers considered that the symptom attention-deficit/hyperactivity disorder were most worrisome. Both series also reported sleep disturbance. Recently, anxiety has been reported in individuals with DDX3X variants. We strongly suggest that attention-deficit/hyperactivity disorder, anxiety, and sleep disorders need to be treated.

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)0.021
metaresearch head score (Gemma)0.120
Version: metacan-v3-hybrid-931329e0061cValidation status: machine_predicted_unvalidated
Candidate categoriesnone
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Observational · Consensus signal: Observational
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.021
Threshold uncertainty score0.113

Distilled classifier scores by category (both heads)

CategoryCodexGemma
Metaresearch0.0210.120
Meta-epidemiology (narrow)0.0010.000
Meta-epidemiology (broad)0.0010.001
Bibliometrics0.0030.002
Science and technology studies0.0010.001
Scholarly communication0.0020.003
Open science0.0020.003
Research integrity0.0020.002
Insufficient payload (model declined to judge)0.0020.001

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 head0.008
GPT teacher head0.253
Teacher spread0.245 · 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 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

Citations5
Published2024
Admission routes2
Has abstractyes

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