Spinal Muscular Atrophy (SMA)
Also known as: Spinal Muscular Atrophies / SMA - Spinal Muscular Atrophy / Muscular Atrophy, Spinal / Spinal Muscular Atrophy / SMA / SMAS / Spinal muscular atrophy, unspecified / Spinal amyotrophy / Spinal muscular atrophy (disorder) / Structure of superficial musculoaponeurotic system (body structure) / Superficial Musculoaponeurotic System
| Drug | Drug Name | Drug Description |
|---|---|---|
| DB13161 | Nusinersen | A medication used to treat spinal muscular atrophy. |
| DB15528 | Onasemnogene abeparvovec | A gene therapy used to treat neonatal and infant patients with spinal muscular atrophy caused by bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. |
| DB15305 | Risdiplam | An oral mRNA splicing modifier used in the treatment of spinal muscular atrophy (SMA). |