SMA · Neurology · 3 drugs · 3 indications
| Drug | Company | Mechanism | Modality | Route | Stage |
|---|---|---|---|---|---|
| Emugrobart | ROG.SW | Anti-Myostatin/Activin | Antibody | SC | PHASE3 |
| Zolgensma | NVS | SMN1 gene replacement | Gene therapy | IV | APPROVED |
| Evrysdi | ROG.SW | SMN2 splicing modifier | SMN2 splicing modifier | Oral | APPROVED |