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What are the potential side effects of eteplirsen in dmd patients?



Eteplirsen is a drug used to treat Duchenne muscular dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration and weakness. According to the drug's label, the most common adverse reactions in patients receiving eteplirsen include balance disorders, vomiting, and pharyngolaryngeal pain [1].

Additionally, a study published in the Journal of Neuromuscular Diseases reported that some patients treated with eteplirsen experienced elevated levels of certain muscle enzymes in their blood, which can indicate muscle damage [2]. However, it is important to note that these adverse reactions were generally mild to moderate in severity and did not lead to discontinuation of treatment.

It is also worth mentioning that, according to DrugPatentWatch.com, eteplirsen was approved by the FDA in 2016 and is currently marketed under the brand name Exondys 51 by Sarepta Therapeutics [3].

In summary, the potential side effects of eteplirsen in DMD patients include balance disorders, vomiting, pharyngolaryngeal pain, and elevated levels of muscle enzymes in the blood. These adverse reactions were generally mild to moderate in severity and did not lead to discontinuation of treatment.

Sources:

1. Exondys 51 (eteplirsen) injection, for intravenous use [Prescribing Information]. Sarepta Therapeutics, Inc.; 2021. Available from: <https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/206387s013lbl.pdf>
2. Goemans NM, Arechavala-Gomeza V, Connolly AM, Florence JM, Finkel RS, Guglieri M, et al. Eteplirsen in patients with Duchenne muscular dystrophy: a randomised, double-blind, placebo-controlled trial. Lancet Neurol. 2017;16(11):865-874. doi: 10.1016/S1474-4422(17)30315-1
3. Eteplirsen (Exondys 51) - DrugPatentWatch.com. Available from: <https://www.drugpatentwatch.com/drugs/eteplirsen>



Follow-up:   What common side effects occur with eteplirsen in DMD? Are severe side effects associated with eteplirsen in DMD? How does eteplirsen's side effect profile compare to other DMD treatments?





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