How much does Eteplirsen cost? How to buy?

Eteplirsen is a drug used to treat amyotrophic lateral sclerosis (DuchenneALS, DMD ). DMD is a rare genetic disorder that primarily affects men and causes progressive muscle weakness and damage. The disease is caused by a genetic mutation on chromosome X that causes the body to fail to produce enough of a protein called "dystrophin" resulting in muscle damage.

Tiplesen has not yet been launched in China, so patients cannot purchase it domestically. There are only American original drugs available abroad, and there are no generic drugs. The price is around 1,600 US dollars, but it is difficult to purchase.

Tiplesen belongs to a class of drugs called "exon skipping enhancer drugs", which work by promoting the expression of the dystrophin gene to alleviate the symptoms of DMD patients. What's special about it is that by promoting specific exon skipping in the dystrophin gene, it enables cells to produce a protein similar to normal dystrophin. Although this protein may still be inferior to normal dystrophin, it helps reduce patients' symptoms and improve their quality of life.

Tiplesen is usually given as an intravenous injection under the supervision of a professional physician. However, it is not a treatment for all patients with DMD. Doctors will decide whether and how to use tiplisen based on the patient's genetic mutation, disease status and individual circumstances.