Spinraza

Spinraza is the brand name for nusinersen, an antisense oligonucleotide therapy approved for spinal muscular atrophy (SMA). It is designed to increase production of functional SMN protein by modifying the splicing of the SMN2 gene, which normally produces insufficient amounts of SMN protein in SMA.

Administration and dosing are intrathecal, delivered directly into the spinal canal. The recommended regimen begins with four loading doses on day 0, day 14, day 28, and day 63, followed by maintenance injections every four months. Each dose contains 12 mg of nusinersen. Because intrathecal delivery can be challenging in some patients, procedures are performed in specialized clinical settings with monitoring for procedure-related adverse events.

Indications and clinical evidence indicate that Spinraza can improve motor function and milestones, slow disease progression, and, in some cases, improve survival or reduce respiratory complications. Benefits have been observed across various SMA types and ages, including infants and older children and adults in real-world and trial settings.

Safety and monitoring: common adverse events include respiratory infections, constipation, back pain, and headaches related to intrathecal administration. Procedure-related risks such as lumbar puncture–associated complications can occur. Serious adverse events are uncommon but can include meningitis or neural injury. Spinraza is contraindicated in individuals with a known hypersensitivity to nusinersen or any excipients in the formulation.

Regulatory status: Spinraza has been approved by major regulatory authorities worldwide and is available in many countries for SMA treatment.