Vyondys 53 (Golodirsen) for Muscular dystrophy

Overview
Vyondys 53, approved by the U.S. Food and Drug Administration (FDA), is used to treat Duchenne muscular dystrophy (DMD) in people with a confirmed mutation (variation) of the DMD gene that allows for exon 53 skipping. This treatment received accelerated FDA approval based on evidence that it increases production of dystrophin (a muscle protein) in skeletal muscle, which is deficient in people with DMD. Continued approval may depend on further clinical trials demonstrating clear clinical benefits. Vyondys 53 is also known by its drug name, golodirsen.

Vyondys 53 belongs to a class of medications called antisense oligonucleotides, which work by targeting and altering RNA (genetic material) to promote the production of dystrophin. This increase in dystrophin can help strengthen and stabilize muscle fibers, potentially slowing the progression of muscle degeneration in DMD.

How do I take it?
Prescribing information states that Vyondys 53 is administered as an intravenous infusion over 35 to 60 minutes, typically once a week. This infusion requires dilution before administration. People should have their kidney function tested before starting treatment, as well as regular monitoring during treatment. Vyondys 53 should be administered exactly as prescribed by a health care provider.

Side effects
Common side effects of Vyondys 53 include headache, fever, falls, abdominal pain, nasal and throat inflammation (nasopharyngitis), cough, vomiting, and nausea.

Rare but serious side effects may include hypersensitivity reactions such as anaphylaxis (severe allergic reaction), rash, fever, pruritus (itching), urticaria (hives), dermatitis, and exfoliation (skin peeling). Additionally, kidney toxicity may occur; therefore, kidney function should be monitored closely, as creatinine levels may not always reliably indicate kidney issues in people with DMD.

For more information about this treatment, visit:

Label: Vyondys 53 — Golodirsen Injection — DailyMed

Vyondys 53 — Drugs.com