1258984-36-9

Nusinersen(1258984-36-9) is a prescription medication used to treat adult and paediatric spinal muscular atrophy (SMA) by improving peripheral muscle function.SMA is caused by a mutation in a gene called SMN1, which encodes for a protein important for motor neuron survival (survival motor neuron or SMN). Although a nearly identical gene (SMN2) serves as a backup to SMN1, it produces a shorter, less stable protein that does not fully compensate for the loss of the full-length protein normally produced by the SMN1 gene. In contrast, Nusinersen is an antisense oligonucleotide agent that modifies the pre-messenger RNA splicing of the SMN2 gene, thereby facilitating increased production of full-length SMN proteins^[1-2].

Nusinersen(1258984-36-9) can be used to treat adult and paediatric spinal muscular atrophy (SMA) and is also able to improve respiratory function and slow the decline in lung function in paediatric SMA^[1].

[1] MICHELLE A FARRAR; Matthew C K. Treating adults with spinal muscular atrophy with nusinersen.[J]. Journal of Neurology, Neurosurgery, and Psychiatry, 2020. DOI:10.1136/jnnp-2020-324036.
[2] ARCHANA CHACKO. Effect of nusinersen on respiratory function in paediatric spinal muscular atrophy types 1-3.[J]. Thorax, 2022. DOI:10.1136/thoraxjnl-2020-216564.