nusinersen (Spinraza)
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Indications
- treatment of spinal muscular atrophy
Dosage
- intrathecal injection
- 12 mg (5 mL) per administration
- 5 ML nusinersen 2.4 MG/ML Injection [Spinraza]
- 4 loading doses;
- the 1st 3 loading doses at 14-day intervals
- the 4th loading dose 30 days after the 3rd dose
- maintenance dose every 4 months[1]
- treatment expected for lifetime[1]
Adverse effects
Mechanism of action
- SMN2-directed antisense oligonucleotide
Notes
- $750,000 for 1st year, $375,000/year thereafter[1] (drug only, not including intrathecal procedure costs)
More general terms
References
- ↑ Jump up to: 1.0 1.1 1.2 1.3 Gerrity MS, Prasad V, Obley AJ Concerns About the Approval of Nusinersen Sodium by the US Food and Drug Administration. JAMA Intern Med. Published online April 30, 2018 PMID: https://www.ncbi.nlm.nih.gov/pubmed/29710299 https://jamanetwork.com/journals/jamainternalmedicine/fullarticle/2678835
- ↑ HIGHLIGHTS OF PRESCRIBING INFORMATION SPINRAZA (nusinersen) injection, for intrathecal use http://www.accessdata.fda.gov/drugsatfda_docs/label/2016/209531lbl.pdf