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casimersen (Amondys 45)
Indications:
- Duchenne muscular dystrophy with mutation amenable to exon 45 skipping
Dosage:
- intravenous infusion 30 mg/kg
Adverse effects:
- glomerulonephritis
Mechanism of action:
- an antisense oligonucleotide
- increase dystrophin production in skeletal muscle
- clinical benefit, including improved motor function, has not been established
Notes:
- orphan drug
General
oligonucleotide
metabolic agent (metabolic modifier)
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