Exondys 51 (eteplirsen) is a medication used to treat Duchenne muscular dystrophy (DMD) in patients with a specific gene mutation. Dosage Exondys 51 is administered through intravenous (IV) infusion at a dose of 30 mg/kg once weekly. Demographic Exondys 51 is approved for patients with DMD who have a specific gene mutation, known as a "confirmed mutation of the dystrophin gene amenable to exon 51 skipping." The medication is only effective for individuals with this specific mutation, which represents approximately 13% of DMD patients. Uses Exondys 51 is used to treat DMD, a genetic disorder that causes progressive muscle weakness and wasting. DMD is caused by a lack of the protein dystrophin, which results in muscle deterioration and eventual loss of function. Exondys 51 helps to increase the production of dystrophin in individuals with the specific gene mutation targeted by the drug. Side Effects The most common side effects reported with Exondys 51 are headache, nausea, fever, dizziness, and cough. More serious side effects may include hypersensitivity reactions, renal toxicity, and decreased renal function. Interactions Exondys 51 has no known drug interactions and does not affect cytochrome P450 enzymes. Generic Options Currently, there are no generic options for Exondys 51. In conclusion, Exondys 51 is a medication used to treat DMD in patients with a specific gene mutation. It is administered through IV infusion and helps to increase the production of dystrophin in individuals with the targeted mutation. Although it has some common side effects, there are currently no known drug interactions. However, generic options for this medication are not available. It is important to consult with a healthcare professional to determine if Exondys 51 is an appropriate treatment option for individuals with DMD and the specific gene mutation amenable to exon 51 skipping.