What are the side effects of Eteplirsen?

Eteplirsen, an antisense oligonucleotide, is a medication used primarily for the treatment of Duchenne Muscular Dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration and weakness. The drug works by targeting exon 51 of the dystrophin gene, promoting the production of a functional form of the dystrophin protein. While Eteplirsen offers potential benefits for patients with DMD, it is essential to be aware of the possible side effects associated with its use.

One of the most commonly reported side effects of Eteplirsen is headache. Patients undergoing treatment may experience mild to moderate headaches. These headaches are generally manageable with over-the-counter pain relievers and tend to diminish over time as the body adjusts to the medication.

Another frequently observed side effect is injection site reactions. Since Eteplirsen is administered via intravenous infusion, patients might experience pain, swelling, redness, or bruising at the injection site. These reactions are typically mild and transient, often resolving without the need for medical intervention.

Gastrointestinal issues, including nausea, vomiting, and diarrhea, have also been reported among patients treated with Eteplirsen. These symptoms are usually mild to moderate in severity and can be managed with supportive care and dietary adjustments. Ensuring adequate hydration and avoiding foods that may exacerbate gastrointestinal discomfort can be helpful.

Some patients may experience upper respiratory tract infections while on Eteplirsen therapy. Symptoms can include a runny nose, sore throat, cough, and congestion. These infections are generally mild and self-limiting, resolving with standard symptomatic treatment. However, it is important for patients and caregivers to monitor these symptoms and seek medical advice if they worsen or persist.

Fatigue is another side effect that has been noted in patients receiving Eteplirsen. This can range from mild to moderate tiredness and may impact daily activities. Adequate rest, a balanced diet, and engaging in light physical activity as tolerated can help manage fatigue.

Although less common, some patients may experience dizziness while on Eteplirsen. This can usually be managed by avoiding sudden movements and maintaining good hydration. Patients should inform their healthcare provider if dizziness persists or interferes with their daily activities.

In rare cases, Eteplirsen may lead to more serious side effects, such as hypersensitivity reactions. Symptoms of a hypersensitivity reaction can include rash, itching, hives, difficulty breathing, and swelling of the face, lips, tongue, or throat. If any of these symptoms occur, it is crucial to seek immediate medical attention.

It is also important to note that long-term safety data for Eteplirsen is still being collected. Patients receiving this treatment should be regularly monitored by their healthcare provider to assess for any emerging side effects or potential complications.

In conclusion, while Eteplirsen offers promising benefits for patients with Duchenne Muscular Dystrophy, it is associated with a range of side effects that vary in severity. Common side effects include headaches, injection site reactions, gastrointestinal issues, upper respiratory tract infections, fatigue, and dizziness. Rare but serious side effects, such as hypersensitivity reactions, require prompt medical attention. Continuous monitoring and communication with healthcare providers are essential to ensure the safe and effective use of Eteplirsen.