casimersen (Amondys 45)
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Indications
- Duchenne muscular dystrophy with mutation amenable to exon 45 skipping
Dosage
- intravenous infusion 30 mg/kg
Adverse effects
Mechanism of action
- an antisense oligonucleotide
- increase dystrophin production in skeletal muscle
- clinical benefit, including improved motor function, has not been established
Notes
More general terms
References
- ↑ George J Another Duchenne Treatment Gets FDA Nod - Casimersen is third approved RNA therapy for DMD MedPage Today February 25, 2021 https://www.medpagetoday.com/neurology/generalneurology/91367
FDA News Release. Feb 25, 2021 FDA Approves Targeted Treatment for Rare Duchenne Muscular Dystrophy Mutation. https://www.fda.gov/news-events/press-announcements/fda-approves-targeted-treatment-rare-duchenne-muscular-dystrophy-mutation-0