User:Mr. Ibrahem/Eteplirsen

Eteplirsen, sold under the brand name Exondys 51, is a medication used to treat Duchenne muscular dystrophy (DMD) with specific mutations. These specific mutations are present in about 14% of DMD cases. Meaningful benefit in this group; however, is unclear. It is given by injection into a vein.

Common side effects include problems with balance, vomiting, and contact dermatitis. Other health problems may include allergic reactions. It is a form of antisense therapy which results in exon 51 skipping.

Eteplirsen was approved for medical use in the United States in 2016. Approval was so controversial that two FDA review panel members resigned in protest. It use was not approved in Europe due to insufficient evidence. In the United States the cost for a child weighting 20 kg is about 524,000 USD per year.