New Nusinersen Drug Delivery Method Identified for Spinal Muscular Atrophy Patients

STRASBURG, PA- A new report has identified an alternative method to deliver nusinersen to patients with spinal muscular atrophy (SMA) using a subcutaneous intrathecal catheter system (SIC) configured by connecting an intrathecal catheter to an implantable infusion port. SMA is a devastating genetic disease that leads to progressive degeneration of motor neurons that control movement, swallowing, and breathing. It is the leading genetic cause of infant death worldwide. Nusinersen is the first FDA approved therapy for SMA but must be administered into the cerebrospinal fluid by repeat lumbar puncture every 4 months for life. Unfortunately, the majority of surviving SMA patients have skeletal deformities or spinal hardware that make it difficult to safely and reliably access the cerebrospinal fluid. The study, by clinicians and researchers at the Clinic for Special Children in Strasburg,PA and the Nemours/A.I. duPont Hospital for Children in Wilmington DE, appears in the Journal of Pediatric Orthopaedics.


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