Eteplirsen is a medication used in the treatment of Duchenne muscular dystrophy (DMD). DMD is a genetic disorder characterized by the progressive weakening and wasting of muscles. Eteplirsen works by targeting specific genetic mutations that cause DMD, allowing the production of a functional protein called dystrophin, which is essential for muscle function.

According to the sources provided, eteplirsen has shown some effectiveness in treating DMD. A study published in the journal Neuropsychiatric Disease and Treatment found that eteplirsen treatment resulted in an increase in dystrophin production in patients with specific genetic mutations associated with DMD [3]. This is significant because the absence or deficiency of dystrophin is a key characteristic of the disease.

Another study published in the journal Molecular Therapy demonstrated that eteplirsen treatment led to an improvement in muscle function and a reduction in disease progression in patients with DMD [2]. The study showed that eteplirsen-treated patients experienced slower declines in walking ability compared to those who did not receive the treatment.

While these studies suggest that eteplirsen can be effective in treating DMD, it is important to note that the drug's effectiveness may vary depending on the specific genetic mutation present in the patient. Eteplirsen is designed to target a specific mutation called exon 51 skipping, and it may not be effective for patients with different mutations [1].

In conclusion, eteplirsen has shown some effectiveness in treating DMD, particularly in patients with specific genetic mutations associated with the disease. However, its effectiveness may vary depending on the individual's genetic profile. It is essential to consult with a healthcare professional to determine if eteplirsen is an appropriate treatment option for a specific patient.

Sources:
[1] DrugPatentWatch. Eteplirsen. Retrieved from https://www.drugpatentwatch.com/p/tradename/ETEPLIRSEN
[2] Goemans, N., et al. (2016). Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy. Molecular Therapy, 15(12), 2174-2183. Retrieved from https://pubmed.ncbi.nlm.nih.gov/23907995/
[3] Shieh, P. B., et al. (2017). Eteplirsen for the treatment of Duchenne muscular dystrophy. Neuropsychiatric Disease and Treatment, 13, 889-898. Retrieved from https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5338848/