Spinraza

Spinraza (nusinersen) is the first FDA-approved medication (2016) used to treat spinal muscular atrophy (SMA), a rare neuromuscular disorder. Administered via intrathecal injection into the spine, it increases SMN protein production to improve muscle function. Common side effects include respiratory infections, constipation, and injection-site reactions, with risks of low platelets and bleeding. Medscape Reference

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Key Information about Spinraza (Nusinersen):

• What it is used for: Treatment of SMA in pediatric and adult patients.
• Mechanism of Action: It is an antisense oligonucleotide that modifies the splicing of the SMN2 pre-mRNA to increase the production of full-length SMN protein, which is deficient in SMA patients.
• Administration: It is administered by a healthcare professional directly into the central nervous system (intrathecal injection).
• Side Effects: Common adverse reactions include lower respiratory infection, constipation, pneumonia, and headache. It also carries risks of kidney toxicity and coagulation abnormalities, requiring regular lab tests.
• Cost: Spinraza is a very high-cost medication, often costing several hundred thousand dollars per year, with prices varying based on the loading dose and maintenance doses, typically covered by insurance or patient assistance programs