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Published reports of novel oligonucleotides to treat individual patients
| Drug Name | Specificity | Target and Condition | Reference |
|---|---|---|---|
| Milasen | Variant-specific | CLN7 Batten disease | 4 |
| Jacifusen* | Gene-specific | FUS amyotrophic lateral sclerosis | 5 |
| Afinersen* | Gene-specific | C9ORF72 amyotrophic lateral sclerosis | 6 |
| Atipeksen | Variant-specific | ATM ataxia telangectasia | 7 |
A single patient was treated but the mechanism is not specific to that individual.
