Neuromuscular · Rare · teaser
Updated August 2026 · SMN1 (5q)
Teaser case. Selected price and peak-sales fields are held for a paid Brief — the rest is the method.
By phase
Classify
| Asset | Sponsor | Phase | Modality | Population | Signal |
|---|---|---|---|---|---|
| Muscle-directed (myostatin / troponin) Residual weakness | Several | Phase 2 | Biologic | Treated SMA with residual deficit | held |
| Higher-dose nusinersen SMN2 splice | Biogen | Phase 3 | ASO / exon skip | SMA on or naive to Spinraza | — |