Spinal muscular atrophy, or SMA, is a rare condition that affects the muscles used for movement, breathing, and everyday activities. In children with SMA, the nerves that control muscles don’t work as well, which can cause muscle weakness that may change over time.
There are several types of SMA, and symptoms can range from mild to more serious. Some children may have trouble walking or climbing stairs, while others may need help with breathing or eating. Early diagnosis and treatment can make a big difference, and many children are now living longer, healthier lives thanks to new therapies.

At Children’s Wisconsin Neurosciences Center, we provide comprehensive care for children with SMA, including the latest FDA-approved treatments, personalized care plans, and access to cutting-edge clinical trials that give families hope and options for the future.
 

Spinal muscular atrophy (SMA) can look different in every child. In the past, SMA was described using four “types” based on the age when symptoms started and how severe they were. Today, with earlier diagnosis and new treatment options, specialists focus more on how and when symptoms begin and on creating personalized care plans for each child.
While every child’s journey is unique, SMA is often grouped more broadly by when symptoms appear:

SMA in infancy: Some babies show signs of muscle weakness very early, such as trouble holding up their head, moving their arms or legs or meeting motor milestones. With today's treatments, many infants are now reaching milestones that were not plausible in the past.

SMA in childhood: Some children develop symptoms later, such as difficulty walking, climbing stairs or getting up from the floor. Muscle weakness may progress over time and some children may need mobility supports.

SMA later in life: In some cases, symptoms don't appear until the teen years or adulthood, and weakness tends to progress more slowly. 

Thanks to major advances in therapies and the addition of SMA to the Newborn Screening Panel in Wisconsin, many children with SMA are living longer, stronger, and more active lives than ever before. At the Children’s Wisconsin Neurosciences Center, we provide comprehensive care and access to the latest treatments and clinical trials, helping families navigate each stage of the journey.

At the Children’s Wisconsin Neurosciences Center, we provide comprehensive, personalized care for children with spinal muscular atrophy (SMA). Treatment plans are tailored to your child’s unique needs and may depend on factors such as:

• Your child's age, overall health and medical history
• How and when symptoms began
• Your child's tolerance for certain medications, procedures or therapies
• Your family's goals and preferences 

While there is no cure for SMA today, there has been tremendous progress in available therapies. At Children’s Wisconsin, we offer the latest FDA-approved treatments and access to cutting-edge clinical trials that are changing the outlook for children with SMA:

• Gene replacement therapy (such as Zolgensma): A one time treatment that targets the genetic cause of SMA for eligible infants and young children
• Antisense oglinucleotide therapies (such as Spinraza): An infusion-based treatment designed to help improve muscle strength and slow disease progression
• Oral SMA therapies (such as Evrysdi): A medication taken at home that can benefit children and adults with certain forms of SMA
• Access to leading-edge clinical trials: Providing families the opportunity to explore new therapies before they are widely available

In addition to medical treatments, our multidisciplinary neuromuscular team provides coordinated care to support your child’s overall health and quality of life. This includes physical and occupational therapy, respiratory care, nutritional guidance, and support from specialists who help families navigate every aspect of living with SMA.

Most advanced program in the state: At the Children’s Wisconsin Neurosciences Center, we provide comprehensive, family-centered care for children with spinal muscular atrophy (SMA). As the largest and most advanced pediatric neurosciences program in the state, we offer families access to the latest therapies, expert specialists, and cutting-edge clinical trials — all in one place.

Expert multidisciplinary team: SMA affects more than just muscle strength, so our care team brings together specialists in neuromuscular medicine, pulmonology, cardiology, genetics, physical therapy, rehabilitation, nutrition, and social work. We work closely with families to design personalized treatment plans that address every aspect of your child’s care and quality of life.

•  Gene replacement therapy for eligible infants and young children
• Infusion and oral therapies designed to improve strength and slow disease progression
• Access to innovative research studies that give families more options than ever before

National recognition: Our Neurosciences Center is consistently ranked among the nation’s best by U.S. News & World Report. Families across the region turn to us for state-of-the-art care, compassionate support, and hope for the future.

Comprehensive support for families: Beyond medical care, we provide resources to support your child’s development and well-being, including rehabilitation services, respiratory care, and community support programs. We also connect families with organizations like the Muscular Dystrophy Association (MDA) for additional education and advocacy.

At the Children’s Wisconsin Neurosciences Center, we know that receiving a diagnosis of spinal muscular atrophy (SMA) can feel overwhelming — but today, there is more hope than ever before. With advances in newborn screening and treatment options, we are able to diagnose and treat SMA earlier and more effectively than ever.

Early Diagnosis Through Newborn Screening
SMA is now included on Wisconsin’s newborn screening panel, which means most babies are diagnosed before symptoms even appear. If your baby’s newborn screen suggests SMA, our team will see you within 24 to 48 hours to confirm the diagnosis using genetic testing and begin treatment planning immediately.

A New Era of Early Treatment
Research has shown that starting treatment before symptoms develop offers the best chance for stronger motor skills and better long-term outcomes. For infants who qualify, we work quickly to assess eligibility for urgent gene replacement therapy and other advanced treatment options.

Comprehensive Evaluation
For children diagnosed later or with symptoms already present, your visit may include:

• A detailed review of your child's medical history and development
• A physical exam focused on muscle strength, breathing and motor skills
• Genetic testing to confirm the diagnosis — now the gold standard for SMA
• Additional assessments by our multidisciplinary team, including pulmonology, cardiology, physical therapy and nutrition specialists

Support for Your Family
From the moment of diagnosis, we provide personalized care and guidance, connecting families with our dedicated SMA care team, educational resources and supportive community programs.