EXONDYS 51 100mg / 2ml solution for injection medication leaflet

M09AX06 • eteplirsen • Musculo-skeletal system | Other drugs for disorders of the musculo-skeletal system

Eteplirsen is a medicine used for selected people with Duchenne muscular dystrophy, a rare genetic disorder that gradually causes muscle weakness. It is only relevant when genetic testing confirms a dystrophin gene mutation that is amenable to exon 51 skipping, so molecular diagnosis is essential before treatment is considered.

Eteplirsen is designed to help muscle cells make a shorter, partly functional form of dystrophin, a protein that helps protect muscle fibres. It does not cure Duchenne muscular dystrophy or fully restore damaged muscle, but it may be part of a broader care plan that includes neurology, heart and lung monitoring, physiotherapy and nutrition.

The medicine is given as an intravenous infusion, usually once weekly, in a specialist setting or as arranged by the care team. The dose is calculated from body weight. Infusions should not be stopped or delayed without medical advice, and caregivers should tell the team about infections, hospital admissions, planned procedures or any major change in strength, breathing or general condition.

Possible side effects include headache, cough, skin rash, vomiting, nausea, dizziness or infusion-related symptoms. Seek urgent medical help if wheezing, chest pain, hives, swelling of the face, fast heartbeat or severe breathing difficulty occurs. Regular follow-up may include kidney checks and assessment of muscle, respiratory and cardiac function.