New Product - Spinraza
Spinraza (nusinersen (heptadecasodium)) is an antisense oligonucleotide specifically designed to treat spinal muscular atrophy (SMA), an autosomal recessive progressive neuromuscular disease, due to mutations in the chromosome 5q. These mutations lead to loss of function of the survival motor neuron 1 (SMN1) gene, resulting in deficiency of SMN protein. The SMN2 gene also produces SMN protein but at low levels. Nusinersen modulates alternate splicing of the SMN2 gene, thus increasing the level of SMN protein in the CNS. Spinraza is indicated for the treatment of 5q SMA. Spinraza solution for intrathecal injection (nusinersen 12 mg) is available in a single 5 mL vial.
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