How Does Spinraza Treatment Work?
Spinraza, also known as nusinersen, is a prescription medicine used to treat Spinal Muscular Atrophy in both pediatric and adult patients. SMA is connected to a mutated or missing SMN1 gene, while the backup SMN2 gene does not naturally produce enough full-length SMN protein for motor neurons to survive.
Spinraza works by targeting the SMN2 gene and helping it produce more complete survival motor neuron protein, which is the protein people with SMA need for motor neuron function. Spinraza is an SMN-enhancing therapy that targets the SMN2 gene, causing it to make more complete protein. In simpler terms, Spinraza does not replace the missing SMN1 gene; instead, it helps the body use its backup gene more effectively. Nusinersen changes SMN2 pre-RNA splicing so exon 7 is included, increasing functional SMN protein.
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