Search
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
General
metabolic agent (metabolic modifier)
References
- Gerorge J
FDA Greenlights New Duchenne Drug - Golodirsen, rejected in August,
is now approved.
MedPage Today. Dec 13, 2019
https://www.medpagetoday.com/neurology/generalneurology/83873