Nusinersen

Nusinersen is a medication used to treat spinal muscular atrophy (SMA), a genetic disorder that affects muscle strength and movement. SMA occurs due to a deficiency of a protein called survival motor neuron (SMN), which is essential for the health of nerve cells that control muscles. Nusinersen works by increasing the production of this protein, helping to improve muscle function and mobility in patients with SMA. It is administered through injections into the spinal canal, and while it cannot cure the disease, it can significantly enhance the quality of life for those affected by SMA.