Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy

This study evaluates the ThecaFlex DRx intrathecal port and catheter system for children and adults with spinal muscular atrophy (SMA) who need repeated spinal injections of nusinersen (Spinraza), a drug that treats SMA by modifying RNA splicing to increase functional motor neuron protein levels. Nusinersen must be injected directly into the spinal canal every four months for life, but in SMA patients, spinal deformities often make standard lumbar punctures extremely difficult, requiring anesthesia, radiation, and complex procedures each time. Patients aged 3 years and older with SMA who are candidates for nusinersen treatment and for whom standard lumbar puncture is difficult or high-risk due to respiratory issues, scoliosis, or other factors are eligible. Patients with recent intrathecal devices, certain spinal abnormalities, active infections, coagulation disorders, hydrocephalus, history of meningitis within six months, degenerative muscular diseases other than SMA, or limited life expectancy are excluded. Participants have the port surgically implanted and receive their scheduled nusinersen doses through the port, with safety and access outcomes tracked over time. This research is vital because reliable drug delivery is fundamental to SMA treatment effectiveness, and a safe implanted access system could transform the treatment experience for patients with the most challenging anatomy.