Second gene therapy for hemophilia B approved: More answers or questions?
Bibliographic record
Abstract
Hemophilia A (hemA) and hemB are X-linked recessive bleeding disorders resulting from low or absent coagulation factor VIII (FVIII) or FIX activity, respectively. FVIII or FIX deficiency leads to debilitating or fatal bleeding episodes if untreated or inadequately treated. Hemophilia was to be the paradigm of Ted Friedmann’s vision of a cure for monogenic diseases through the transfer of replacement genes.1 Three products are now approved for hemophilia gene therapy, two for hemB and one for hemA, with possible approval of a second FVIII product in the next year.
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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 | 0.010 | 0.020 |
| Meta-epidemiology (narrow) | 0.003 | 0.001 |
| Meta-epidemiology (broad) | 0.004 | 0.003 |
| Bibliometrics | 0.002 | 0.001 |
| Science and technology studies | 0.003 | 0.003 |
| Scholarly communication | 0.007 | 0.004 |
| Open science | 0.004 | 0.001 |
| Research integrity | 0.028 | 0.030 |
| Insufficient payload (model declined to judge) | 0.012 | 0.007 |
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".