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Record W2138366034 · doi:10.1517/14656566.9.2.311

Idursulfase for the treatment of mucopolysaccharidosis II

2008· review· en· W2138366034 on OpenAlexaffabout
L. Clarke

Bibliographic record

VenueExpert Opinion on Pharmacotherapy · 2008
Typereview
Languageen
FieldMedicine
TopicLysosomal Storage Disorders Research
Canadian institutionsChild and Family Research Institute
Fundersnot available
KeywordsHunter syndromeEnzyme replacement therapyRecombinant DNAMedicineMucopolysaccharidosisMucopolysaccharidosis type IIDiseaseGlycosylationGenetic enhancementBioinformaticsInternal medicineBiologyGeneticsGene

Abstract

fetched live from OpenAlex

Human recombinant proteins are being used to treat an increasing number of disorders. Advances in the large scale production of recombinant proteins and the understanding of glycosylation and its importance for protein targeting and function have led to the development of recombinant enzyme-replacement regimens for a number of human lysosomal storage diseases. The latest addition to this group is mucopolysaccharidosis II or Hunter syndrome. Purified human recombinant idursulfase has been shown to alter disease manifestations in individuals with Hunter syndrome. The recent approval in the US, Europe, Canada and Japan of idursulfase for the treatment of Hunter syndrome introduces the first pharmacologic agent and indeed the first specific treatment directed towards this devastating genetic disease.

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 categoriesMeta-epidemiology (narrow)
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Not applicable · Consensus signal: none
GenreCandidate signal: Review · Consensus signal: Review
Teacher disagreement score0.960
Threshold uncertainty score1.000

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0000.000
Meta-epidemiology (narrow)0.0010.000
Meta-epidemiology (broad)0.0020.002
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)0.0010.000

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 head0.178
GPT teacher head0.486
Teacher spread0.309 · 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.

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

Citations23
Published2008
Admission routes2
Has abstractyes

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