Nusinersen is an antisense oligonucleotide (ASO) drug used to treat spinal muscular atrophy (SMA).
- Basic information
- English name: Nusirersen
- CAS number: 1258984-36-9
- Molecular formula: C ₂∝₄ H ∝₂∝ N ₆₁ O ₁₂₈ P ₁₉ S ₁₉
- Molecular weight: ≈ 7501.6 g/mol
- Structure type: Thiophosphate modified antisense oligonucleotides (20 mer)
- Indications: Used for the treatment of 5q type spinal muscular atrophy (SMA) (including infantile, pediatric, and adult types).
- Mechanism of Action
Nusirersen is an antisense oligonucleotide (ASO) that functions through the following mechanisms:
- Targeting SMN2 gene: SMA patients have a deficiency of motor neuron survival protein (SMN) due to SMN1 gene deletion/mutation.
- Promoting complete splicing of SMN2 gene: Under normal circumstances, SMN2 gene produces truncated non functional SMN protein (about 90%), while Nusirersen can bind to SMN2 pre mRNA, promoting the preservation of its exon 7 (Exon 7), thereby increasing the production of full-length functional SMN protein and improving motor neuron function.
- Clinical efficacy
- For infantile SMA (SMA1): significantly prolonged survival and improved motor function (such as increased CHOP-INTEND score).
- For late-onset SMA (SMA2/3): Improve muscle strength and exercise ability (such as increasing Hammersmith Motor Function Scale scores).
- Long term efficacy: Continuous treatment can maintain or further improve motor function.
Product Manager Email: ada@coreychem.com
