Delandistrogene moxeparvovec

Delandistrogene moxeparvovec is used to treat Duchenne muscular dystrophy in children who are 4 to 5 years old and have a specific gene mutation.

The doctor will confirm that your child has the right gene mutation to receive delandistrogene moxeparvovec.

Delandistrogene moxeparvovec may also be used for other reasons not mentioned in this medication guide.

Delandistrogene moxeparvovec can lead to serious side effects (even weeks or months after your child has received this treatment). Inform your child's doctor if they experience: chest pain, shortness of breath, difficulty speaking or swallowing, increased muscle pain, weakness, tenderness, jaundice (yellowing of the skin or eyes), or cold symptoms.

Your child should not receive delandistrogene moxeparvovec if they have a specific type of gene mutation. Ask your child's doctor about this risk.

Tell your child's doctor if they have or have ever had:

• recently had or have an infection;
• a history of high levels of a specific type of antibody called anti-AAVrh74;
• heart problems; or
• liver disease.

Make sure your child is up to date on all vaccines at least 4 weeks before starting delandistrogene moxeparvovec.

Get emergency medical help if you notice signs of an allergic reaction: hives, trouble breathing, swelling of your face, lips, tongue, or throat.

Delandistrogene moxeparvovec can lead to serious side effects (even weeks or months after your child has received this treatment). Contact your child's doctor immediately if they experience:

• liver problems--loss of appetite, nausea, vomiting, stomach pain (upper right side), tiredness, itching, dark urine, clay-colored stools, jaundice (yellowing of the skin or eyes);
• cold symptoms--runny nose, sneezing, sore throat, fever, cough;
• chest pain, shortness of breath; or
• difficulty speaking or swallowing, increased muscle pain, weakness or tenderness.

Common side effects of delandistrogene moxeparvovec may include:

• fever;
• low levels of platelets in your blood;
• vomiting, nausea; or
• abnormal liver function tests.

This is not a complete list of side effects, and others may happen. Contact your doctor for medical advice about side effects. You can report side effects to the FDA at 1-800-FDA-1088.

Your child's doctor will perform tests before starting treatment with delandistrogene moxeparvovec to make sure it is the right treatment for your child.

Doses are based on weight. Your doctor will confirm your child's weight before starting treatment with delandistrogene moxeparvovec.

Delandistrogene moxeparvovec is given in one dose and injected into a vein with an infusion pump. A healthcare provider will give this injection.

Your child will need frequent blood tests before and after receiving delandistrogene moxeparvovec.

Your child will be given other medications by mouth before and after receiving delandistrogene moxeparvovec to help prevent serious side effects. Your child must keep taking these medicines for as long as the doctor has prescribed. Tell the doctor right away if your child misses a dose or vomits shortly after taking these medications.

The child's caregiver should practice proper hand hygiene when coming into direct contact with the child's body waste for 1 month after the child receives delandistrogene moxeparvovec. Place any materials that may have your child's body waste in a sealed plastic bag and dispose in regular trash.

Delandistrogene moxeparvovec is given as one dose and does not have a set dosing schedule.

In a medical setting, an overdose would be handled promptly.

Follow your child's doctor's instructions regarding any limits on food, drinks, or activities.

Other drugs may affect delandistrogene moxeparvovec, including prescription and over-the-counter medicines, vitamins, and herbal products. Inform your doctor about all other medicines you are using.