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Spinraza to treat spinal muscular atrophy

Spinraza is a treatment for spinal muscular atrophy (SMA). The drug, known generically as nusinersen, was approved by the Federal Drug Administration (FDA) in 2016. It is the only medication approved by the FDA to treat SMA.

Since its approval, Children’s Mercy has worked with families to evaluate if Spinraza is an option for their child. The families who have chosen to begin the treatment have reported significant improvement in their child’s condition and well-being. 

Success when using Spinraza will mean something different for each child. Some have found they stay awake longer during the day, are more vocal, or have the strength to lift their head when they could not before. Other children have seen even more dramatic results. Some can now feed themselves and others have regained the ability to walk.

Our team is based out of Rehabilitation, but works alongside staff members from several departments in the hospital. Each team member works closely with families so they understand the benefits of using Spinraza as well as the costs of the treatment. 

What is spinal muscular atrophy?

Spinal muscular atrophy is progressive and eventually leads to poor muscle tone, weakness, an inability to swallow, and respiratory failure. Symptoms typically appear before six months of age and are accompanied by feeding and breathing difficulties. It affects approximately 1 in 10,000 people.

Prior to Spinraza, only supportive care was available to children with SMA.

To learn more about SMA, visit Cure SMA.

How does Spinraza work?

Spinal muscular atrophy is caused by mutations in the SMN1 gene and its severity is affected by extra copies of the SM2 gene. Spinraza works by increasing the body’s ability to produce SMN protein, which helps create healthier and stronger motor neurons. The treatment was approved by the FDA for people of all ages affected by SMA and ongoing clinical trials continue to demonstrate its effectiveness.

What is the process to be treated with Spinraza?

We evaluate each child to see if they are a good candidate for Spinraza, as each child’s condition is different. If we determine Spinraza could be a beneficial treatment, we begin the pre-authorization process with your insurance. We have a team that specializes in authorization specifically for Spinraza. In the meantime, our clinical team will begin working with your family on lab work and other details.

How is Spinraza administered?

Spinraza is given by injection in the lower back. The drug is injected into the spinal fluid around the spinal cord. Remaining very still during the procedure is important, so some children may require sedation or general anesthesia during the procedure to help with this.

Your child will receive four initial injections in the first two months of treatment. They will then receive maintenance injections every four months after that. Prior to each administration of treatment, our multidisciplinary team will review your child’s health to get a full integrated picture of how Spinraza is working.

What is the cost of Spinraza?

We often receive questions about how much Spinraza costs. It is important to discuss Spinraza with your insurance provider, as coverage is different under every plan. Children’s Mercy works with every family to carefully navigate pre-authorization, billing and insurance.