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

More general terms

• oligonucleotide
• metabolic agent (metabolic modifier)