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What is Spinraza (nusinersen) for?
Spinraza (nusinersen) is indicated for the treatment of pediatric and adult patients with spinal muscular atrophy (SMA)[1,4].
How does Spinraza (nusinersen) work?
SMA is a rare and often fatal hereditary (genetic) disease that causes weakness and muscle wasting due to the loss of motor neurons controlling movement.
Spinraza (nusinersen) is an antisense oligonucleotide (ASO) that is designed to treat SMA caused by mutations in the chromosome 5q. This mutation leads to the deficiency of a protein, the survival motor neuron (SMN) protein. Spinraza (nusinersen) alters the synthesis of the deficient protein to increase production of full-length SMN protein and thereby promoting the maintenance of motor neurons.
How do I take Spinraza (nusinersen)?
Spinraza is an injection administered into the fluid surrounding the spinal cord (intrathecal injection)[2,5]. The standard dosage is:
- 12 mg / 5 mL per administration
Common side effects of Spinraza (nusinersen)
Side effects include:
- back pain