User:Mr. Ibrahem/Viltolarsen

Viltolarsen, sold under the brand name Viltepso, is a medication used to treat certain cases of Duchenne muscular dystrophy (DMD). Specifically it is used for the approximately 8% of people with DMD who have a mutation that is amenable to exon 53 skipping. As of 2020 it was unclear if it results in improvements. It is given by injection into a vein over an hour.

Common side effects include upper respiratory tract infection, injection site reaction, cough, and fever. Other potential concerns include kidney problems. It is a morpholino antisense oligonucleotide.

Viltolarsen was approved for medical use in the United States in 2020. It is not approved in Europe, as of 2020, though has orphan designation. In the United States it costs about 1,500 USD per 250 mg as of 2021. This is about 730,000 USD for a person who is 30 kg.