Spinal Muscular Atrophy (SMA) in children

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What is spinal muscular atrophy?

Spinal muscular atrophy (SMA) is a rare genetic condition in which muscles throughout the body are weakened because cells in the spinal cord and brainstem do not work properly. SMA is the number one genetic cause of infant mortality.

There are three types of SMA:

  • Type I is the most severe form of SMA. It’s sometimes called Werdnig-Hoffmann disease. Children with type I have limited movement, can’t sit without support, and have trouble breathing, feeding and swallowing. Symptoms begin within months after birth. Many children with type I do not live past age 2.
  • Type II is an intermediate form of SMA. Children with this type may sit without support at some point but cannot walk on their own. Symptoms typically start between 6 and 18 months of age. Depending on the severity of symptoms, children with type II may have a normal life span. 
  • Type III is a milder form of SMA. It’s also known as Kugelberg-Welander disease and resembles muscular dystrophy (MD). While children with type III usually are able to walk, most have some difficulty walking. Some may eventually need to use a wheelchair. Symptoms usually appear around 18 months of age or in early childhood. Children with this type of SMA generally have an almost normal life expectancy. 

Treatment of SMA consists mainly of managing the symptoms, providing respiratory support as needed and preventing disease complications.

Care for spinal muscular atrophy

The Spinal Muscular Atrophy Program at Boston Children’s Hospital brings together a team of experts from different specialties who are experienced in caring for children with SMA.

Our program also gives parents a chance to speak with other families facing similar challenges with SMA.

We are grateful to have been ranked #1 on U.S. News & World Report's list of the best children's hospitals in the nation for the third year in a row, an honor we could not have achieved without the patients and families who inspire us to do our very best for them. Thanks to you, Boston Children's is a place where we can write the greatest children's stories ever told.”
- Sandra L. Fenwick, President and CEO

Boston Children's Hospital
300 Longwood Avenue, Boston, MA 02115
For Patients: 617-355-6000 | 800-355-7944
For Referring Providers: 844-BCH-PEDS

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