Golodirsen

From WikiMD.org
Jump to navigation Jump to search

Golodirsen (pronounced go-lo-DIR-sen) is a medication used in the treatment of Duchenne muscular dystrophy (DMD) in certain patients. It is marketed under the brand name Vyondys 53 by Sarepta Therapeutics.

Etymology

The name "Golodirsen" is derived from the drug's chemical structure. It is a morpholino antisense oligomer that induces exon 53 skipping in the dystrophin gene.

Pharmacology

Golodirsen works by binding to exon 53 of the dystrophin pre-mRNA, resulting in exclusion, or "skipping," of this exon during mRNA processing. Skipping exon 53 allows for the production of an internally truncated, but functional form of dystrophin protein in patients with genetic mutations that are amenable to exon 53 skipping.

Usage

Golodirsen is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This group represents about 8% of the total DMD population.

Side Effects

Common side effects of Golodirsen include headache, fever, fall, abdominal pain, nasopharyngitis, cough, vomiting, and nausea. Serious side effects may include hypersensitivity reactions and kidney damage.

Related Terms

External links

Esculaap.svg

This WikiMD dictionary article is a stub. You can help make it a full article.


Languages: - East Asian 中文, 日本, 한국어, South Asian हिन्दी, Urdu, বাংলা, తెలుగు, தமிழ், ಕನ್ನಡ,
Southeast Asian Indonesian, Vietnamese, Thai, မြန်မာဘာသာ, European español, Deutsch, français, русский, português do Brasil, Italian, polski