Nusinersen is an antisense oligonucleotide active molecule. Nusinersen modifies the pre-messenger RNA splicing of the SMN2 gene, thereby promoting the production of full-length SMN protein. Nusinersen improves spinal muscular atrophy.
Products | Products > Disease Areas > Neurological, Eye or Ear Disease | Products > Signaling Pathways > Cell Cycle/DNA Damage | Products > Research Areas > Neurological Disease | Products > Signaling Pathways > DNA/RNA Synthesis
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