Spinraza® (nusinersen)
for Spinal Muscular Atrophy
Spinraza (nusinersen) is an antisense oligonucleotide medication used to treat children and adults with spinal muscular atrophy (SMA), a motor neuron disease caused by mutations in the SMN1 gene. This leads to a lack of functional SMN protein, which is vital for motor neuron health. Spinraza works by modifying the SMN2 gene to include the missing information from exon 7, ensuring the produced SMN protein functions properly, improving motor neuron function and muscle strength.
