Spinraza

Spinraza (nusinersen) is a medication used to treat spinal muscular atrophy (SMA), a genetic disorder that causes weakness and loss of muscle mass due to missing or defective survival motor neuron (SMN) protein. It is an antisense oligonucleotide that is administered via injections into the spinal canal, helping to increase production of SMN protein in nerve cells. This improvement can slow or stop the progression of muscle weakness, allowing individuals with SMA to maintain mobility and function longer. Spinraza represents a significant advancement in managing this challenging condition by addressing its underlying cause.