Patient demographics and clinical characteristics of adults with SMA: assessment of risdiplam-treated and untreated SMA
Bibliographic record
Abstract
<h3></h3> The efficacy of risdiplam has been demonstrated in clinical trials that included adult patients, but the real-world effectiveness of risdiplam in adults has not yet been extensively investigated This non-interventional, retrospective cohort study aims to describe adult SMA patients in terms of their demographic and clinical characteristics, treatment patterns and healthcare resource utilisation. If possible, it will also explore how newly treated SMA adults receiving risdiplam compare with patients who are not receiving DMTs. This study will use data from seven registries (clinician and patient reported) from within the TREAT-NMD network (Australia, Belgium, Canada, Czech Republic & Slovakia, Germany & Austria, Ukraine, UK and Ireland) to identify adult SMA patients treated with risdiplam and compare with an untreated patient population, in registries deemed feasible for a comparative analysis. Appropriate adjustment methods will be explored to adjust for potential confounders, and appropriate statistical models will be used to perform comparisons with untreated patients. The analysis of this study is ongoing as of the submission of this abstract. Available data from the comparative analyses will be presented. This study will provide additional evidence to further support the understanding of treatment effectiveness in adults with SMA.
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 imitationNot 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.
Codex and Gemma teacher scores by category
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.000 | 0.000 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.000 | 0.000 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.000 | 0.000 |
| Insufficient payload (model declined to judge) | 0.000 | 0.000 |
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.
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 teacher head, 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".