How is Spinraza administered?

Spinraza (nusinersen) is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide used to treat spinal muscular atrophy (SMA) in adults and children.

SMA is a type of motor neuron disease that destroys motor neurons - the muscle-controlling nerve cells. It is caused by a mutation in the SMN1 (survival motor neuron 1) gene, which is responsible for producing the SMN protein that keeps motor neurons healthy and functioning.

Spinraza is administered by injection at a hospital or an outpatient site with the necessary experience.

How does Spinraza come and how is it administered?

Spinraza comes in a single-use vial and is injected using a syringe directly into the cerebrospinal fluid (CSF), which is the fluid around the spine and brain. It is given over 1-3 minutes. This type of injection is called an intrathecal bolus injection.

Spinraza is administered by healthcare professionals who are experienced in performing lumbar punctures.

Spinraza needs to be delivered directly into the central nervous system (CNS) because of the way it works - it's mechanism of action.

How often is Spinraza taken?

Four loading doses of Spinraza are given when treatment is started. The first three loading doses are given at 14 day intervals and the fourth is given 30 days after the third loading dose. A maintenance does of Spinraza is then given once every four months.