Spinraza (nusinersen)

Spinraza (nusinersen) is a medication used to treat spinal muscular atrophy (SMA), a genetic condition that weakens muscles and affects movement. It works by increasing the production of a vital protein called survival motor neuron (SMN), which is lacking in people with SMA. Spinraza is administered via injections into the spinal canal, allowing it to reach the nervous system directly. By boosting SMN levels, it helps improve muscle strength and function, potentially stabilizing or slowing the progression of the disease. It is the first approved treatment specifically targeting the root cause of SMA.