What is Eteplirsen?

Eteplirsen (Eteplirsen) is a drug used to treat Duchenne muscular dystrophy (Duchennemuscular dystrophy), developed by Sarepta Therapeutics. Duchenne muscular dystrophy is a rare genetic disease that primarily affects males and usually begins to develop in childhood. The disease causes patients' muscles to gradually weaken and atrophy, ultimately affecting their ability to exercise and quality of life.

Tiplesen is a type of exon skipping therapy (exon-skipping therapy) and is a gene therapy method. In people with Duchenne muscular dystrophy, certain exons (gene segments) are missing from the gene, causing problems with the production of muscle proteins that trigger symptoms of the disease. Tiplerisen's mechanism is to interfere with this genetic defect, prompting cells to skip the missing exon, so that the gene transcription process can produce relatively normal muscle proteins.

Specifically, Tiplerisen injects the drug into the patient's muscle, acting on the precursorRNA (mRNA) of a specific gene, encouraging cells to skip the missing exons, thereby producing a partially functional protein that, although not completely normal, can alleviate muscle damage to a certain extent. This treatment is not a cure, but it can help slow the progression of the disease and improve the patient's quality of life.

Tiplerisen has shown some positive effects in clinical trials, particularly improvements in some patients' ability to walk. However, its effects may vary between patients. Because this is a newer treatment, its long-term effects are still being studied to understand.

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