golodirsen; Vyondys 53

Indications

• Duchenne muscular dystrophy* ;

* 8% of DMD patients have the mutation targeted by golodirsen

Adverse effects

• most common
  • headache, fever, cough, nausea/vomiting, abdominal pain
  • cold symptoms
• hypersensitivity reactions, including rash, fever, itching, hives, dermatitis, skin exfoliation
• nephrotoxicity seen in animals

Mechanism of action

• exon 53-skipping RNA therapy

Notes

• viltolarsen (Viltepso) also treats DMD patients with the same mutation

More general terms

• metabolic agent (metabolic modifier)