New, Life-Saving Treatment for Genetic Neuromuscular Disorder Used for First Time in New Jersey
Hackensack Meridian Health’s Joseph M. Sanzari Children’s Hospital Successfully Administers Treatment for 20-Month-Old Child with Spinal Muscular Atrophy
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Hackensack Meridian Health’s Joseph M. Sanzari Children’s Hospital at Hackensack University Medical Center became the first hospital in New Jersey to deliver the new potentially life-saving gene therapy treatment, onasemnogene abeparvovec-xioi (Zolgensma®), to a 20-month-old patient with spinal muscular atrophy (SMA) Type 1. The patient was initially treated with nusinersen (Spinraza®) when he was diagnosed at 6 months old.
Having this treatment available in New Jersey will allow the children’s hospital to not only provide cutting-edge care for children with SMA, but to also eliminate the burden, expense, and stress that travel can place on the family during a difficult time if the treatment was not available locally.
New Jersey is close to adding testing for SMA to the newborn screening panel as part of a state-mandated series of genetic tests. As a New Jersey Department of Health-designated newborn screening follow-up center, the children’s hospital provides expertise in the early detection and diagnosis of genetic disorders which leads to better outcomes and more options for treatment.