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Nusinersen is an antisense oligonucleotide compound that modifies pre-messenger RNA splicing of the SMN2 gene, thereby promoting full-length SMN protein production.
| Molecular Weight | 7127.194237 |
| Formula | C234H340N61O128P17S17 |
| CAS Number | 1258984-36-9 |
| Storage | 4-8°C |
[1] Qing Li. Yonsei Med J. Nusinersen as a Therapeutic Agent for Spinal Muscular Atrophy
[2] Melanie K Claborn, et al. Ann Pharmacother. Nusinersen: A Treatment for Spinal Muscular Atrophy
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