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NBTs approved by FDA for CNS disorders *Not commercially available.
| Condition | Drug name | Target | Delivery method | Manufacturer | Year approved |
|---|---|---|---|---|---|
| Spinal muscular atrophy | Nusinersen (Spinraza) | SMN2 | IT | Ionis/Biogen | 2016 |
| Batten’s disease | Milasen* | ATXN2 | IT | Boston Children’s Hospital | 2018 |
| Amyotrophic lateral sclerosis | Tofersem (Qalsody) | SOD1 | IT | Ionis/Biogen | 2023 |
