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nusinersen (Spinraza)
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:
- upper respiratory tract infection
- lower respiratory tract infection
- constipation
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)
General
neurologic agent
oligonucleotide
Database Correlations
PUBCHEM cid=124037382
References
- 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: 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