Spinal Muscular Atrophy Treatments

Children’s Mercy rehabilitation medicine physicians offer cutting-edge treatments for children with spinal muscular atrophy (SMA). This genetic disorder of the nervous system affects the nerve cells in your child’s spinal cord that control movement and muscle strength.

Currently, there are two FDA-approved treatment options for SMA, Spinraza and Zolgensma. Children’s Mercy offers both these treatments for children who qualify.

What is spinal muscular atrophy?

Spinal muscular atrophy is a progressive condition that 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 11,000 people.

Prior to Spinraza, the first medical treatment for SMA, only supportive care was available to children with SMA. Now, both Spinraza and Zolgensma are approved treatments, and several others are in various stages of research and development.

As more states (including Missouri) are beginning to require newborn screenings for SMA, many more children are able to be diagnosed and begin treatment earlier for better outcomes.

To learn more about SMA, visit Cure SMA.

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 one of only two medications 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. Families who have chosen to begin the treatment have reported significant improvement in their child’s condition and wellbeing. 

Success when using Spinraza will mean something different for each child. Some families have found that their child stays awake longer during the day, is more vocal, or has 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.

The SMA team at Children’s Mercy is based out of our Rehabilitation Medicine department but works alongside staff members from several different specialties. The SMA team works closely with families so they understand the benefits of using Spinraza as well as the costs of the treatment. 

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 who are affected by SMA. 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.

Zolgensma for spinal muscular atrophy treatment

Zolgensma is the second medication approved for use by the FDA in children under two years of age with SMA. It is a type of gene therapy, which means the medication treats the genetic cause of SMA by replacing the SMN1 gene.

Zolgensma is administered to your child as a one-time infusion through an IV. The infusion takes about an hour.

Timing and cost of treatment

With both Spinraza and Zolgensma, the sooner your child can receive treatment after their diagnosis, the better the results. It is often possible to begin treatment once your child’s diagnosis is confirmed, even if they have not yet begun to show symptoms of SMA.

Your SMA team at Children’s Mercy will help you determine which treatment is best for your child and navigate the process of pre-authorization, billing and insurance with your family.

Choosing the best home for your child’s care

Children’s Mercy has always been on the leading edge of care for children with SMA. Soon after the FDA approval of Spinraza in December 2016, we launched our SMA Care Team. Since that time, we have treated dozens of children and seen extremely positive results. All of the families who have received treatment at Children’s Mercy so far have reported improvements in their child’s quality of life and none have experienced significant complications from the treatment.