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A patient at Boys Town National Research Hospital was among the first in the United States to receive ITVISMA, a new FDA-approved treatment for spinal muscular atrophy (SMA).
After receiving its first delivery of ITVISMA on April 8, Boys Town Pediatric Neurology became the first site west of the Mississippi River—and the fourth nationwide—to administer the treatment. Boys Town Hospital is working through the insurance authorization process with several other SMA patients so they may also benefit from ITVISMA.
SMA is a neurodegenerative disorder caused by mutations in the survival motor neuron 1 (SMN1) gene. It affects the motor nerve cells in the spinal cord, impacting muscles used for activities like breathing, eating, crawling and walking. It can lead to paralysis and death in the most severe cases. SMA affects approximately 1 in 15,000 live births in the U.S.
A single dose of ITVISMA has been shown in clinical trials to stop SMA progression and improve motor function. As a gene therapy, it works by replacing the function of the missing or nonworking SMN1 gene with a new, working copy of an SMN gene.
ITVISMA is approved for SMA patients 2 years of age and older with a confirmed SMN1 mutation. It offers a gene therapy option for patients who are older than the approved age range for Zolgensma, which is limited to patients under 2 years of age.
Boys Town Pediatric Neurology is committed to offering leading-edge therapies such as ITVISMA and Zolgensma. Our comprehensive approach to care combines expert diagnosis and advanced therapies with wraparound services that ensure each child and family is supported at every stage of their journey.