Spinraza (nusinersen) is an FDA-approved treatment for spinal muscular atrophy (SMA), a rare genetic disorder that leads to muscle weakness and loss of motor function. It is the first and only approved treatment for SMA. Spinraza works by increasing the amount of a protein called SMN (survival of motor neuron) which is lacking in people with SMA. The drug is administered through a spinal tap, where it is directly injected into the cerebrospinal fluid that surrounds the spinal cord. Spinraza is considered a breakthrough in the treatment of SMA, as it has shown to improve motor function and increase survival rates in patients. However, it is not a cure and requires ongoing treatment and monitoring. The drug is manufactured by Biogen.
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