CSC Researchers Evaluate the Benefit of Dual Therapy for Children At Risk for Spinal Muscular Atrophy

LANCASTER COUNTY, PA – In a first-of-its-kind study, researchers compared the efficacy of preventative therapy for spinal muscular atrophy (SMA) between two well-matched study groups, using either gene therapy (onasemnogene abeparvovec) alone or in combination with risdiplam (oral medication) or nusinersen (intrathecal injection) administered before apparent signs of disease emerged. The study included presymptomatic infants with two or three copies of SMN2 at risk for developing SMA type 1 or 2, respectively. SMA is a devastating rare genetic disorder that leads to progressive degeneration of spinal motor neurons that control movement, swallowing, and breathing. The paper was recently published by researchers from the Clinic for Special Children and Children’s Hospital of Philadelphia in the Annals of Clinical and Translational Neurology.

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