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Record W4388732614 · doi:10.1002/ajmg.a.63477

The <scp>8th International RASopathies Symposium</scp>: Expanding research and care practice through global collaboration and advocacy

2023· article· en· W4388732614 on OpenAlexaff
Elizabeth I. Pierpont, Anton M. Bennett, Lisa Schoyer, Beth Stronach, April Anschutz, Sarah C. Borrie, Benjamin Briggs, Emma Burkitt‐Wright, Pau Castel, Ion Cristian Cirstea, Fieke Draaisma, Michelle Ellis, Vanessa S. Fear, Megan N. Frone, Elisabetta Flex, Bruce D. Gelb, Tamar Green, Karen W. Gripp, Sattar Khoshkhoo, Mark W. Kieran, Karolin Kleemann, Bonnie Klein-Tasman, Maria Kontaridis, Paul Kruszka, Chiara Leoni, Clifford Z. Liu, Nadia Merchant, Pilar Magoulas, Christopher L. Moertel, Carlos E. Prada, Katherine A. Rauen, Renée L. Roelofs, Rodrigue Rossignol, Christine Sévilla, Gigi Sevilla, Ryan Sheedy, Elliot Stieglitz, Daochun Sun, Dagmar K. Tiemens, Forest M. White, Ellen Wingbermühle, Cordula M. Wolf, Martin Zenker, Grégor Andelfinger

Bibliographic record

VenueAmerican Journal of Medical Genetics Part A · 2023
Typearticle
Languageen
FieldBiochemistry, Genetics and Molecular Biology
TopicProtein Tyrosine Phosphatases
Canadian institutionsMcGill University
FundersCongressionally Directed Medical Research ProgramsNational Cancer InstituteNational Institutes of HealthDeutsche HerzstiftungNational Institute of Neurological Disorders and StrokeBundesministerium für Bildung und ForschungDeutsche ForschungsgemeinschaftRevolution MedicinesBioMarin PharmaceuticalU.S. Department of DefenseEuropean CommissionNational Center for Advancing Translational SciencesChildren's Tumor FoundationChildren's Hospital ColoradoNational Heart, Lung, and Blood InstitutePfizerBoehringer IngelheimAmerican Heart AssociationAlexion PharmaceuticalsDoris Duke Charitable Foundation
KeywordsNoonan syndromeCostello syndromeGeneral partnershipIntellectual propertyShort statureMedicinePolitical scienceCancerPediatricsInternal medicine

Abstract

fetched live from OpenAlex

Germline pathogenic variants in the RAS/mitogen-activated protein kinase (MAPK) signaling pathway are the molecular cause of RASopathies, a group of clinically overlapping genetic syndromes. RASopathies constitute a wide clinical spectrum characterized by distinct facial features, short stature, predisposition to cancer, and variable anomalies in nearly all the major body systems. With increasing global recognition of these conditions, the 8th International RASopathies Symposium spotlighted global perspectives on clinical care and research, including strategies for building international collaborations and developing diverse patient cohorts in anticipation of interventional trials. This biannual meeting, organized by RASopathies Network, was held in a hybrid virtual/in-person format. The agenda featured emerging discoveries and case findings as well as progress in preclinical and therapeutic pipelines. Stakeholders including basic scientists, clinician-scientists, practitioners, industry representatives, patients, and family advocates gathered to discuss cutting edge science, recognize current gaps in knowledge, and hear from people with RASopathies about the experience of daily living. Presentations by RASopathy self-advocates and early-stage investigators were featured throughout the program to encourage a sustainable, diverse, long-term research and advocacy partnership focused on improving health and bringing treatments to people with RASopathies.

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)0.011
metaresearch head score (Gemma)0.013
Version: metacan-v3-hybrid-931329e0061cValidation status: machine_predicted_unvalidated
Candidate categoriesnone
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Not applicable · Consensus signal: Not applicable
GenreCandidate signal: Other · Consensus signal: none
Teacher disagreement score0.046
Threshold uncertainty score0.154

Distilled classifier scores by category (both heads)

CategoryCodexGemma
Metaresearch0.0110.013
Meta-epidemiology (narrow)0.0010.000
Meta-epidemiology (broad)0.0010.001
Bibliometrics0.0010.001
Science and technology studies0.0040.003
Scholarly communication0.0070.004
Open science0.0020.011
Research integrity0.0100.012
Insufficient payload (model declined to judge)0.0460.014

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 head0.021
GPT teacher head0.385
Teacher spread0.364 · 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 designNot applicable
Domainnot available
GenreOther

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

Citations7
Published2023
Admission routes1
Has abstractyes

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Same venueAmerican Journal of Medical Genetics Part ASame topicProtein Tyrosine PhosphatasesFrench-language works237,207