Spinal Muscular Atrophy (SMA): Treatment

If you have questions or are worried about anything, call the Neurogenetics Unit at 617-726-5732. You can also reach the genetic counselor at 617-726-4878.

How do doctors treat spinal muscular atrophy (SMA)?

There is no cure for SMA. Treatment depends on your child’s symptoms and when they start. Treatment works best when it starts as early as possible.

Treatments can include:

• Nusinersen (Spinraza®). This is a medication injected into the spinal fluid. It helps your child’s body make more survival motor neuron protein. Research has shown that Spinraza® can help your child meet milestones and keep the spinal nerves working properly for longer.
• Physical therapy to help strengthen his muscles.
• If your child has trouble breathing, breathing machines (also called respiratory support) or cough assisted machine can help him breathe easier.

Who is on my child's care team?

There are many people on your child’s care team to help make sure he gets the best care possible. Care team members can include:

• Geneticist (doctor who studies genetic conditions)
• Neurologist (doctor who care for problems with the brain and nerves)
• Physical therapist
• Pulmonologist (lung doctor)
• Nutritionist
• Orthopaedics
• Primary care doctor
• Genetic counselor (person who helps you coordinate genetic testing and explains the meaning of the results for your family)

Where can I learn more about SMA?

• Your child's care team
• Cure SMA®
• Genetic and Rare Diseases Information Center (GARD)®
• Genetics Home Reference®
• Muscular Dystrophy Association

Rev. 3/2018. Reviewed by the MGfC Family Advisory Council. Mass General for Children and Massachusetts General Hospital do not endorse any of the brands listed on this handout. This handout is intended to provide health information so that you can be better informed. It is not a substitute for medical advice and should not be used to treatment of any medical conditions.