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 movement2.
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 in order to increase production of full-length SMN protein and thereby promoting the maintenance of motor neurons3.
Is Spinraza (nusinersen) approved?
Spinraza (nusinersen) was approved for the treatment of children and adults with spinal muscular atrophy (SMA) by:
- FDA (USA) on December 23, 20162
- EMA (EU) on May 30, 20175
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
Initiate treatment with 4 loading doses. The first dose should be followed by 3 more doses after 2, 4, and 9 weeks and then one dose every 4 months thereafter. Treatment should be continued for as long as the patient benefits from it1,5.
Complete information about Spinraza (nusinersen) dosage and administration can be found in the resources section.