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Record W2170425676 · doi:10.1586/17446651.2.2.147

Agalsidase alfa therapy for Fabry disease

2007· article· en· W2170425676 on OpenAlexaff
Sandra Sirrs, Joe T.R. Clarke

Bibliographic record

VenueExpert Review of Endocrinology & Metabolism · 2007
Typearticle
Languageen
FieldMedicine
TopicLysosomal Storage Disorders Research
Canadian institutionsCentre Hospitalier Universitaire de SherbrookeHospital for Sick ChildrenStornoway Diamond (Canada)
Fundersnot available
KeywordsFabry diseaseEnzyme replacement therapyMedicineStroke (engine)Left ventricular hypertrophyGenetic enhancementDiseaseLysosomal storage diseaseRenal functionCardiomyopathyCardiologyNeuropathic painInternal medicineEndocrinologyBioinformaticsGenePharmacologyHeart failureBiochemistryBiology

Abstract

fetched live from OpenAlex

Fabry disease is a lysosomal storage disorder that results in neuropathic pain, progressive renal dysfunction, cardiomyopathy and stroke in affected individuals. The disease is caused by mutations in the GLA gene coding for α galactosidase A. The resulting deficiency of this enzyme causes accumulation of neutral glycosphingolipids in various tissues. Recombinant human agalsidase alfa has been developed to treat patients with Fabry disease. Preliminary data on this form of enzyme replacement therapy suggest that it improves pain, stabilizes renal function and improves cardiac hypertrophy in some patients. More data are needed on the ability of this therapy to prevent cardiac events, stroke and death.

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)0.001
metaresearch head score (Gemma)0.001
Version: codex-gemma-dda1882f352aValidation 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 score0.916
Threshold uncertainty score0.864

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0010.001
Meta-epidemiology (narrow)0.0000.000
Meta-epidemiology (broad)0.0010.001
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 head0.036
GPT teacher head0.396
Teacher spread0.360 · 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 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

Citations1
Published2007
Admission routes1
Has abstractyes

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