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 |
| Species | Mouse | Rat | Rabbit | Guinea pig | Hamster | Dog |
| Weight (kg) | 0.02 | 0.15 | 1.8 | 0.4 | 0.08 | 10 |
| Body Surface Area (m2) | 0.007 | 0.025 | 0.15 | 0.05 | 0.02 | 0.5 |
| Km factor | 3 | 6 | 12 | 8 | 5 | 20 |
| Animal A (mg/kg) = Animal B (mg/kg) multiplied by | Animal B Km |
| Animal A Km |
For example, to modify the dose of Compound A used for a mouse (20 mg/kg) to a dose based on the BSA for a rat, multiply 20 mg/kg by the Km factor for a mouse and then divide by the Km factor for a rat. This calculation results in a rat equivalent dose for Compound A of 10 mg/kg.
[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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