SUNFISH: 4-year efficacy and safety data of risdiplam in types 2 and 3 SMA
Bibliographic record
Abstract
<h3></h3> Risdiplam (EVRYSDI®) is an oral survival of motor neuron (<i>SMN2</i>) pre-mRNA splicing modifier approved by the EMA and MHRA for the treatment of patients aged ≥2 months with Type 1, 2 or 3 spinal muscular atrophy (SMA) or 1–4 <i>SMN2</i> copies. SUNFISH (NCT02908685) is a two-part, randomised, placebo-controlled, double-blind study in patients with Types 2/3 SMA aged 2–25 years at enrolment. Part 2 (N=180) assessed efficacy and safety of the Part 1-selected dose of risdiplam versus placebo in Type 2 and non-ambulant Type 3 SMA. Participants were treated with risdiplam or placebo for 12 months, then received risdiplam in a blinded manner until Month 24, when they entered the open-label extension. The primary endpoint (Part 2) of change from baseline in the 32-item Motor Function Measure (MFM32) total score in patients receiving risdiplam (n=120) versus placebo (n=60) was met at Month 12. Motor function increases were sustained in the second and third year after risdiplam treatment (assessed by the MFM32, Hammersmith Functional Motor Scale – Expanded and Revised Upper Limb Module). At Month 36, no safety findings led to treatment withdrawal in SUNFISH Part 1 or 2. Here we present 4-year efficacy and safety data from SUNFISH.
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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".