Search
viltolarsen (Viltepso)
Indications:
- treatment of Duchenne muscular dystrophy with confirmed mutation in the DMD gene amenable to exon 53 skipping (8% of patients)
Dosage:
- weekly infusions over 60 minutes
Adverse effects:
- upper respiratory tract infection
- injection site reaction
- cough
- fever. ;
* glomerulonephritis observed after administration of some antisense oligonucleotides
Mechanism of action:
- antisense oligonucleotide
Notes:
- golodirsen (Vyondys 53) also treats same DMD gene mutation
General
oligonucleotide
metabolic agent (metabolic modifier)
References
- George J
Rare Duchenne Muscular Dystrophy Mutation Has New Tx -
FDA approves antisense oligonucleotide viltolarsen
MedPage Today August 12, 2020
https://www.medpagetoday.com/neurology/generalneurology/88038