SUNFISH: 4-year efficacy and safety data of risdiplam in types 2 and 3 SMA
Bibliographic record
Abstract
Risdiplam (EVRYSDI®) is an oral survival of motor neuron (SMN2) 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 SMN2 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 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 | 0.003 | 0.002 |
| Meta-epidemiology (narrow) | 0.001 | 0.000 |
| Meta-epidemiology (broad) | 0.002 | 0.003 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.001 |
| Scholarly communication | 0.001 | 0.001 |
| Open science | 0.001 | 0.000 |
| Research integrity | 0.001 | 0.002 |
| Insufficient payload (model declined to judge) | 0.004 | 0.001 |
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".