How Spinal Muscular Atrophy (SMA) Is Treated

Treatments for the genetic disorder spinal muscular atrophy (SMA) include two approaches—disease-modifying therapy and symptom control. Spinraza (nusinersen), Zolgensma (onasemnogene abeparovec-xioi), and Evrysdi (risdiplam) are the three treatments that are approved for preventing SMA from worsening, and they are considered disease-modifying treatments.

In addition to these disease-modifying therapies, other important aspects of managing SMA include at-home approaches such as respiratory care, optimization of motor control with physical therapy, and the use of devices to support the spine and muscles. Sometimes surgery may be needed to manage serious complications of SMA.

Beautiful little boy on wheelchair smiling and looking with admiration at his unrecongizable physical therapist
Hispanolistic / Getty Images

Disease Management 

SMA disease-modifying treatment has advanced substantially with the approval of three treatments: Spinraza in 2016, Zolgensma in 2019, and Evrysdi in 2020. The prognosis and disease progression of SMA may be improved with these treatments. Keep in mind that because these medications are relatively new, the long-term outcome is not yet known.


Spinraza was approved by the U.S. Food and Drug Administration (FDA) in 2016. This medication is taken by intrathecal injection, a procedure in which the medicine is injected directly into the spinal fluid with a needle. This procedure must be done by a trained physician.

Treatments are injected as three loading doses every two weeks, followed by a fourth loading dose after one month. After the four initial loading doses, you would receive an injection of Spinraza every four months.

According to the manufacturer, the recommended dosage of Spinraza is 12 milligrams (mg), or 5 milliliters (mL), per administration.

Spinraza works by stimulating production of the SMN protein, a protein that is deficient in SMA. Replacement of SMN protein with this procedure helps the motor neurons in the body to function properly, preventing the disease from progressing.


Zolgensma was approved by the FDA in 2019 for use in babies under two years old who have infantile-onset SMA.

It is used as a one-time intravenous (IV—injected in a vein) treatment. According to the manufacturer, the recommended dosage of Zolgensma is 1.1 × 1014 vector genomes (vg) per kilogram (kg) of body weight.

This medication is a type of gene therapy. It works by delivering a normal copy of the SMN gene (which is altered in SMA) into the blood through a non-infectious virus. The normal gene replaces the altered SMA gene so the body can produce SMN protein.


Evrysdi (risdiplam) was approved by the FDA in 2020 for adults and children two months and older with SMA. In May 2022, the FDA expanded the usage to treat SMA in all adults and infants.  

It is an oral solution prepared by a healthcare provider and given at home once daily using a syringe. The recommended dosage for children is based on age and weight, while the recommended dosage for adults is 5 mg daily.

Evrysdi is a type of gene therapy. It works by altering the SMN2 gene to stimulate the production of functional SMN protein throughout the body. 

All of these therapies have been studied in SMA, with promising results. You would have to discuss your specific situation with your healthcare provider to decide which therapeutic plan is right for you or your child.

Strategies for managing complications of SMA with at-home approaches, physical therapy, and prescriptions may be necessary even if you use disease-modifying therapy. It is important that you maintain close follow up with your healthcare provider so that any issues that you may develop, such as breathing problems or infections, can be managed promptly.

Home Remedies and Lifestyle

Living with SMA requires lifestyle adjustments that affect the whole family. Often, family members need to learn how to assist with mobility.

Additionally, family members may need to assist with using at-home therapies. Respiratory devices are the most important and frequently used at-home tools in SMA.

Respiratory Devices

There is a range in the types of respiratory care used in managing SMA. Some devices provide oxygen or minimal airway pressure and can be easily used at home. Many people who have mild breathing problems with SMA only need to use breathing assistance while sleeping or during times when breathing is especially difficult, such as during an infection.

If your breathing becomes more impaired as your disease progresses, it may become necessary to advance to increasingly supportive and consistent respiratory care. Machines that provide full breathing assistance can aid with inspiration (breathing in) and expiration (breathing out) when respiratory muscles are very weak. Sometimes oxygen is provided through a respiratory machine as well.

Supportive Splint or Brace

If you or your child has developed musculoskeletal complications such as scoliosis or contractures, a brace or other supportive device may be necessary to hold the body in a comfortable and safe position.

You may need a brace or splint to support your arms, wrist, leg, or ankle. These should be custom fit to your size and needs. Using supportive devices can help you optimize your ability to get around safely and use your limbs (arms and legs).

If you have developed scoliosis, you may need an external back brace to support you and to prevent your spine from curving. With advanced SMA, you may need to secure your position in a wheelchair to prevent your back from leaning to one side, which can exacerbate scoliosis.

Wheelchair or Walker

You may need an assistive device to help you get around. There is a range of support that you can use, depending on your muscle control. Options include a cane, a walker, a wheelchair, or a mobilized wheelchair.

Physical and Occupational Therapy

Often, the effects of the motor limitations of SMA can be improved with interactive physical exercises and rehabilitative therapy approaches.

While physical therapy techniques cannot repair the nerves or muscles or reverse muscle atrophy, you can learn to optimize your motor strength, control, and coordination.

You can also learn to avoid injuries by learning how to control your weight and your limbs as well as possible. Therapy can often help prevent muscle contractures.

Goals and types of therapy used in SMA include:

  • Muscle strengthening and coordination: Consistent physical therapy with a therapist is often combined with exercises that you can learn to do at home. Consistently moving your muscles can help minimize muscle atrophy and maximize muscle control.
  • Swallowing safety: Sometimes choking can be a risk with SMA. Working with a specialist to learn to control swallowing muscles can prevent complications like aspiration pneumonia and choking.
  • Respiratory management: Because the muscles that control breathing are often weakened in SMA, exercises that help you to strengthen and control these muscles can be an important part of managing your condition.

Breathing exercises may include the use of a device at home. You may be instructed to take a certain number of deep breaths every day with a device that measures inspiratory and expiratory volume (how deep you breathe in and out). Your healthcare provider or therapist may work with you to create targets and goals that you can measure with your respiratory device.

Passive muscle movements: If you or your child has severely diminished muscle strength, passive movements may be necessary. A family member or a therapist would move your muscles on a regularly scheduled basis to prevent contractures and pressure sores from developing.

Surgeries and Specialist-Driven Procedures

In some circumstances, surgery may be beneficial for the treatment of complications of SMA. There are several procedures that can help in managing the effects of this condition.

Procedures that may be needed to manage complications of SMA include:

Botulinum toxin: In situations when muscle stiffness is interfering with mobility, injections of botulinum toxin, a powerful muscle relaxant, may help. Often, oral muscle relaxants cause intolerable side effects, while injections can concentrate the effects of the medication towards the areas that need it most, with fewer side effects.

Release of contractures: When muscle contractures are painful or interfere with mobility, a surgical procedure to release contractures can be helpful in some situations.

Spine surgery: Scoliosis can be very severe in SMA, and the bony structural changes can impinge on the chest area, potentially impairing breathing. Spine surgery to surgically repair scoliosis may be necessary for some circumstances. Sometimes a supportive device, such as a rod, is surgically placed near the spine to support it and help keep it straight.

Tracheostomy: In some situations, the most effective way to manage breathing problems in SMA is with the use of a tracheostomy. This is a surgical procedure that involves the placement of a breathing tube directly in the throat. The tube may be attached to a mechanical device that aids with breathing.

Complementary and Alternative Medicine (CAM)

Alternative medicine is not considered an effective approach for disease modification or for preventing the complications of SMA. It is important that you discuss any treatment that you are interested in with your healthcare provider to ensure that you do not expose yourself to anything that is not beneficial or that could be harmful to your overall health.

Frequently Asked Questions

  • How are babies with the SMN1 mutation treated?

    Three gene therapy drugs have been approved since 2016 for children under age 2. These drugs stimulate the production of the SMN protein by a child’s cells so it can begin producing effective proteins that can improve neuron and motor function.

  • Does insurance cover Zolgensma for type 1 SMA treatment?

    Zolgensma is a one-time treatment for type 1 spinal muscle atrophy with a list price of about $2,125,000. Insurance may cover it, but in some instances, your child may need to be symptomatic (versus being diagnosed via genetic testing). The manufacturer offers financial help. Despite the excessively high cost, Zolgensma is considered the most cost-effective treatment for type 1 SMA.

  • Is spinal muscular atrophy curable?

    Unfortunately, no. Treatments can help manage symptoms and stop the disease from getting worse, but there's no cure for SMA. For those diagnosed with type 2, 3, or 4, treatment options will usually enable you to enjoy a normal life expectancy.

11 Sources
Verywell Health uses only high-quality sources, including peer-reviewed studies, to support the facts within our articles. Read our editorial process to learn more about how we fact-check and keep our content accurate, reliable, and trustworthy.
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  8. Rul B, Carnevale F, Estournet B, Rudler M, Hervé C. Tracheotomy and children with spinal muscular atrophy type 1: ethical considerations in the French context. Nurs Ethics. 2012;19(3):408-18. doi:10.1177/0969733011429014

  9. SMA News Today. What Is Spinal Muscular Atrophy?

  10. Dean R, Jensen I, Cyr P, et al. An updated cost-utility model for onasemnogene abeparvovec (Zolgensma®) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and Effectiveness Review (ICER). Journal of Market Access & Health Policy. 2021;9(1):1889841. doi:10.1080%2F20016689.2021.1889841

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Additional Reading

By Heidi Moawad, MD
Heidi Moawad is a neurologist and expert in the field of brain health and neurological disorders. Dr. Moawad regularly writes and edits health and career content for medical books and publications.