Plain Language Summary: Looking at treatment outcomes in people with Fabry disease who started agalsidase beta before the age of 30 years
Bibliographic record
Abstract
What did this study look at? An enzyme called alpha-galactosidase (alpha-Gal A) does not work properly or is absent in people with Fabry disease. Agalsidase beta (an enzyme replacement therapy) is a treatment for Fabry disease that substitutes for the non-working or absent alpha-Gal A enzyme. Using information from the Fabry Registry on young people who started agalsidase beta treatment between 5 and 30 years of age, researchers looked at the following over time: Kidney function decline.Heart muscle health.Fabry-related symptoms: ○ Stomach aches. ○ Diarrhoea. ○ Tingling or burning pain in hands and feet. ○ Pain crises (attacks of severe pain, also called Fabry crises).Characteristics of the people studied were: Males were younger than females when: ○ Symptoms first started. ○ Agalsidase beta treatment was first started.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot 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.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.002 | 0.034 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.002 |
| Bibliometrics | 0.002 | 0.003 |
| Science and technology studies | 0.001 | 0.000 |
| Scholarly communication | 0.001 | 0.002 |
| Open science | 0.001 | 0.001 |
| Research integrity | 0.002 | 0.001 |
| Insufficient payload (model declined to judge) | 0.078 | 0.012 |
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.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
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".