Spinraza is ann intrathecally administered antisense oligonucleotide (ASO) for treatment of spinal muscular atrophy (SMA). SMA is caused by deletions or mutations in the survival motor neuron 1 (SMN1) gene; these gene alterations lead to a deficiency of survival motor neuron (SMN) protein, which is critical for the maintenance of motor neurons. Spinraza is designed to alter the splicing of SMN2, a gene that is nearly identical to SMN1, in order to increase production of fully functional SMN protein. Spinraza is delivered by intrathecal injection, since ASO drugs do not cross an intact blood–brain barrier when delivered systemically. The drug is administered via lumbar puncture with topical anesthesia. 

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