User:Mr. Ibrahem/Golodirsen

Golodirsen, sold under the brand name Vyondys53, is a medication used to treat Duchenne muscular dystrophy (DMD) due to a mutation in the dystrophin gene that may be improved by exon 53 skipping. It is given by injection into a vein. While it increases dystrophin, benefit is yet to be shown as of 2021.

Common side effects include headache, fever, falls, abdominal pain, runny nose, and nausea. Other side effects may include allergic reactions and kidney problems. It is an antisense oligonucleotide that binds to exon 53 and results in mRNA that produces more functional dystrophin.

Golodirsen was approved for medical use in the United States in 2019. As of 2021 it is not approved in either Europe or the United Kingdom. In the United States it costs about 5,000 USD for a 10 kg person per week as of 2021.