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

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.