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Record W2914250213 · doi:10.1002/jimd.12024

International clinical guidelines for the management of phosphomannomutase 2‐congenital disorders of glycosylation: Diagnosis, treatment and follow up

2019· review· en· W2914250213 on OpenAlexaff
Ruqaiah Altassan, Romain Péanne, Jaak Jaeken, Rita Barone, Muad Bidet, Delphine Borgel, Sandra Brasil, David Cassiman, Anna Čechová, David Coman, Javier Corral, Joana Correia, María Eugenia de la Morena‐Barrio, Pascale de Lonlay, Vanessa dos Reis, Carlos R. Ferreira, Agata Fiumara, Rita Francisco, Hudson H. Freeze, Simone Funke, Thatjana Gardeitchik, Gert Matthijs, Muriel Girad, M. Girós, Stephanie Grünewald, Trinidad Hernández‐Caselles, Tomáš Honzík, Marlen Hutter, Donna M. Krasnewich, Christina Lam, Joy Lee, Dirk J. Lefeber, Dorinda Marques‐da‐Silva, Antonio Federico Martínez‐Monseny, Hossein Moravej, Katrin Õunap, Carlota Pascoal, Tiffany Pascreau, Marc C. Patterson, Dulce Quelhas, Kimiyo Raymond, Peymaneh Sarkhail, Manuel Schiff, Małgorzata Seroczyńska, Mercedes Serrano, Nathalie Séta, Jolanta Sykut‐Cegielska, Christian Thiel, Federic Tort, Mari‐Anne Vals, Paula A. Videira, Peter Witters, Renate Zeevaert, Éva Morava

Bibliographic record

VenueJournal of Inherited Metabolic Disease · 2019
Typereview
Languageen
FieldBiochemistry, Genetics and Molecular Biology
TopicGlycosylation and Glycoproteins Research
Canadian institutionsMontreal Children's Hospital
Fundersnot available
KeywordsGuidelinePresentation (obstetrics)MedicineIntensive care medicinePediatricsMultidisciplinary approachClinical PracticePathologySurgeryPhysical therapy

Abstract

fetched live from OpenAlex

Phosphomannomutase 2 (PMM2-CDG) is the most common congenital disorder of N-glycosylation and is caused by a deficient PMM2 activity. The clinical presentation and the onset of PMM2-CDG vary among affected individuals ranging from a severe antenatal presentation with multisystem involvement to mild adulthood presentation limited to minor neurological involvement. Management of affected patients requires a multidisciplinary approach. In this article, a systematic review of the literature on PMM2-CDG was conducted by a group of international experts in different aspects of CDG. Our managment guidelines were initiated based on the available evidence-based data and experts' opinions. This guideline mainly addresses the clinical evaluation of each system/organ involved in PMM2-CDG, and the recommended management approach. It is the first systematic review of current practices in PMM2-CDG and the first guidelines aiming at establishing a practical approach to the recognition, diagnosis and management of PMM2-CDG patients.

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)0.002
metaresearch head score (Gemma)0.007
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: none
GenreCandidate signal: Review · Consensus signal: Review
Teacher disagreement score0.008
Threshold uncertainty score0.027

Distilled classifier scores by category (both heads)

CategoryCodexGemma
Metaresearch0.0020.007
Meta-epidemiology (narrow)0.0010.000
Meta-epidemiology (broad)0.0020.002
Bibliometrics0.0050.003
Science and technology studies0.0010.001
Scholarly communication0.0010.001
Open science0.0030.001
Research integrity0.0030.002
Insufficient payload (model declined to judge)0.0080.004

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 head0.131
GPT teacher head0.443
Teacher spread0.312 · 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
GenreReview

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

Citations148
Published2019
Admission routes1
Has abstractyes

Explore more

Same venueJournal of Inherited Metabolic DiseaseSame topicGlycosylation and Glycoproteins ResearchFrench-language works237,207