← Mechanisms of action
SMN2 pre-mRNA
NUCLEIC-ACIDHomo sapiens
Drugs acting on this target (5)
| Active Pharmaceutical Ingredient | Mechanism Type ▲ | Approval Status | API Type | Mechanism of Action |
|---|---|---|---|---|
| biib115 | Antisense inhibitor | experimental | Nucleic acid | SMN2 pre-mRNA antisense inhibitor |
| nusinersen (Spinraza) | Modulator | approved | Small molecule | SMN2 pre-mRNA modulator |
| ro6885247 | Modulator | experimental | Small molecule | SMN2 pre-mRNA modulator |
| nusinersen (Spinraza) | Positive modulator | approved | Small molecule | SMN2 pre-mRNA positive modulator |
| risdiplam (Evrysdi) | Positive modulator | approved | Small molecule | SMN2 pre-mRNA positive modulator |