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A New Life Saving Drug for SMA - Pediatric Neurology

“It is an absolutely devastating disorder,” pediatric neurologist Dr. Britt Stroud with Golisano Children’s Hospital of Southwest Florida, is referring to spinal muscular atrophy, or SMA for short. “It’s the most common infant cause of genetic death, but it affects about one in ten kids world-wide.” The disease affects the motor nerve cells in the spinal cord—making children unable to walk, eat, or breathe. Studies show only 8 percent of children diagnosed with SMA live to 20 months old. “The goal is to try to save those motor neurons, so rescue those motor neurons before they die,” he explained. And a new FDA approved medication called Zolgensma is doing just that. “For those patients who are found to have SMA, they now have a choice to replace that defective gene with another human SMA one gene with a one-time IV dose,” said Dr. Stroud. Children with SMA typically have low tone, weakness, and loss of reflexes. If children have SMA and are under the age of 2, they may qualify for zolgensma. “It’s a very fast-acting mechanism. This rescues those motor neurons and provides life,” he said. Golisano Children’s Hospital is the only hospital in Southwest Florida, offering the gene replacement therapy. “We want to bring the highest level of care for SMA patients to Golisano for the children of Southwest Florida,” Dr. Stroud said. Offering families hope and treatment to a devastating diagnosis. View More Health Matters video segments at LeeHealth.org/Healthmatters/ Lee Health in Fort Myers, FL is the largest network of health care facilities in Southwest Florida and is highly respected for its expertise, innovation and quality of care. For more than 100 years, we’ve been providing our community with personalized preventative health services and primary care to highly specialized care services and robotic assisted surgeries. Lee Health - Caring People. Inspiring Care. Visit LeeHealth.org