This information from Lexicomp® explains what you need to know about this medication, including what it’s used for, how to take it, its side effects, and when to call your healthcare provider.
Brand Names: US
Emflaza
What is this drug used for?
- It is used to treat Duchenne muscular dystrophy (DMD).
What do I need to tell the doctor BEFORE my child takes this drug?
- If your child is allergic to this drug; any part of this drug; or any other drugs, foods, or substances. Tell the doctor about the allergy and what signs your child had.
- If your child has an infection. This includes bacterial, viral, and fungal infections; amoeba infection (like traveler’s diarrhea); herpes infection of the eye; malaria infection in the brain; threadworm infestation; or any other infection.
- If your child has any of these health problems: Diverticulitis, hole in the GI (gastrointestinal) tract, stomach or bowel infection, or ulcers.
- If your child has recently had stomach or bowel surgery.
- If your child takes any drugs (prescription or OTC, natural products, vitamins) that must not be taken with this drug, like certain drugs that are used for HIV, infections, seizures, and others. There are many drugs that must not be taken with this drug.
This is not a list of all drugs or health problems that interact with this drug.
Tell the doctor and pharmacist about all of your child’s drugs (prescription or OTC, natural products, vitamins) and health problems. You must check to make sure that it is safe to give this drug with all of your child’s other drugs and health problems. Do not start, stop, or change the dose of any drug your child takes without checking with the doctor.
What are some things I need to know or do while my child takes this drug?
All products:
- Tell all of your child’s health care providers that your child is taking this drug. This includes your child’s doctors, nurses, pharmacists, and dentists.
- Have your child’s blood work checked as you have been told by the doctor. Your child may also need to have eye pressure and bone density checked if this drug is used for a long time.
- Long-term use may raise the chance of cataracts, glaucoma, or weak bones (osteoporosis). Talk with your child’s doctor.
- High blood pressure has happened with this drug. Have your child’s blood pressure checked as you have been told by the doctor.
- If your child has high blood sugar (diabetes), you will need to watch your child’s blood sugar closely.
- You may need to lower how much salt is in your child’s diet and give your child extra potassium. Talk with your child’s doctor.
- Avoid giving your child grapefruit and grapefruit juice.
- If your child is not up to date with vaccines, talk with your child’s doctor. Your child may need to have some vaccines before treatment with this drug.
- Talk with the doctor before your child gets any vaccines. Use of some vaccines with this drug may either raise the chance of very bad infection or make the vaccine not work as well.
- Chickenpox and measles can be very bad or even deadly in some people taking steroid drugs like this drug. Avoid having your child near anyone with chickenpox or measles if your child has not had these health problems before. If your child has been exposed to chickenpox or measles, talk with the doctor.
- Steroid drugs, including this one, raise the risk of infection. They can cause an active infection to get worse. They can also make some signs of infection harder to notice. Infections that happen with steroids can be mild, but they can also be severe and even deadly. The risk of problems from these infections is usually raised with higher doses of steroids. Have your child wash hands often and stay away from people with infections, colds, or flu. Tell the doctor if your child has any signs of an infection.
- Some infections may become active again in patients taking drugs like this one. This includes tuberculosis and hepatitis B. Tell the doctor if your child has ever had one of these infections.
- This drug may lower how much natural steroid is in your child’s body. If your child has a fever, an infection, surgery, or is hurt, talk with the doctor. Your child may need extra doses of oral steroids. These extra steroids will help your child’s body deal with these stresses. Carry a warning card saying that there may be times when your child needs extra steroids.
- Very bad and sometimes life-threatening health problems can happen with drugs like this one, especially with long-term use. This includes changes in adrenal gland function and a tumor on the adrenal gland (pheochromocytoma). Talk with the doctor.
- Blood clots have happened with this drug. Tell the doctor if your child has ever had a blood clot.
- A type of cancer (Kaposi’s sarcoma) has happened in people taking drugs like this one for a long time. Talk with the doctor.
- Do not stop giving this drug to your child all of a sudden without calling the doctor. Your child may have a greater risk of side effects. If your child needs to stop this drug, you will want to slowly stop it as told by the doctor.
- Tell the doctor if your child has missed a dose or recently stopped this drug and feels very tired, weak, or shaky, or has a fast heartbeat, confusion, sweating, or dizziness.
- This drug may affect growth in children and teens in some cases. They may need regular growth checks. Talk with the doctor.
If your child is pregnant or breast-feeding a baby:
- Talk with the doctor if your child is pregnant, becomes pregnant, or is breast-feeding a baby. You will need to talk about the benefits and risks to your child and the baby.
Liquid (suspension):
- This drug has benzyl alcohol in it. If possible, avoid products with benzyl alcohol in newborns or infants. Serious side effects can happen in these children with some doses of benzyl alcohol, including if given with other drugs that have benzyl alcohol. If you have questions, talk with the doctor.
What are some side effects that I need to call my child’s doctor about right away?
WARNING/CAUTION: Even though it may be rare, some people may have very bad and sometimes deadly side effects when taking a drug. Tell your child’s doctor or get medical help right away if your child has any of the following signs or symptoms that may be related to a very bad side effect:
- Signs of an allergic reaction, like rash; hives; itching; red, swollen, blistered, or peeling skin with or without fever; wheezing; tightness in the chest or throat; trouble breathing, swallowing, or talking; unusual hoarseness; or swelling of the mouth, face, lips, tongue, or throat.
- Signs of infection like fever, chills, very bad sore throat, ear or sinus pain, cough, more sputum or change in color of sputum, pain with passing urine, mouth sores, or wound that will not heal.
- Signs of high blood sugar like confusion, feeling sleepy, unusual thirst or hunger, passing urine more often, flushing, fast breathing, or breath that smells like fruit.
- Signs of Cushing’s syndrome like weight gain in the upper back or belly, moon face, severe headache, or slow healing.
- Signs of a weak adrenal gland like a severe upset stomach or throwing up, severe dizziness or passing out, muscle weakness, feeling very tired, mood changes, decreased appetite, or weight loss.
- Signs of high blood pressure like very bad headache or dizziness, passing out, or change in eyesight.
- Signs of electrolyte problems like mood changes; confusion; muscle pain, cramps, or spasms; weakness; shakiness; change in balance; an abnormal heartbeat; seizures; loss of appetite; or severe upset stomach or throwing up.
- Shortness of breath, a big weight gain, or swelling in the arms or legs.
- Skin changes (pimples, stretch marks, slow healing, hair growth).
- Change in eyesight.
- Bone or joint pain.
- Black, tarry, or bloody stools.
- Throwing up blood or throw up that looks like coffee grounds.
- Mental, mood, or behavior changes that are new or worse.
- Purple, red, blue, brown, or black bumps or patches on the skin or in the mouth.
- Call the doctor right away if your child has signs of a blood clot like chest pain or pressure; coughing up blood; shortness of breath; swelling, warmth, numbness, change of color, or pain in a leg or arm; or trouble speaking or swallowing.
- A very bad skin reaction (toxic epidermal necrolysis) may happen. It can cause very bad health problems that may not go away, and sometimes death. Get medical help right away if your child has signs like red, swollen, blistered, or peeling skin (with or without fever); red or irritated eyes; or sores in the mouth, throat, nose, or eyes.
What are some other side effects of this drug?
All drugs may cause side effects. However, many people have no side effects or only have minor side effects. Call your child’s doctor or get medical help if any of these side effects or any other side effects bother your child or do not go away:
- Weight gain.
- Increased appetite.
- Signs of a common cold.
- Nose and throat irritation.
- Passing urine more often.
- Stomach pain.
- Constipation.
- Back pain.
- Upset stomach.
- Headache.
These are not all of the side effects that may occur. If you have questions about side effects, call your child’s doctor. Call your child’s doctor for medical advice about side effects.
You may report side effects to your national health agency.
How is this drug best given?
Give this drug as ordered by your child’s doctor. Read all information given to you. Follow all instructions closely.
Tablets:
- Give this drug with or without food.
- Have your child swallow whole with a full glass of water.
- The tablet may also be crushed and mixed with applesauce.
- After mixing, give the dose right away. Do not store for future use.
- Keep giving this drug to your child as you have been told by your child’s doctor or other health care provider, even if your child feels well.
Liquid (suspension):
- Give this drug with or without food.
- If the adapter has not been put in the bottle when you get it, follow how to prepare the bottle as you have been told or read the package insert.
- Shake well before use.
- Measure liquid doses carefully. Use the measuring device that comes with this drug. If there is none, ask the pharmacist for a device to measure this drug.
- Slowly add the liquid to 3 to 4 ounces (90 to 120 mL) of juice or milk. Mix well. Do not mix with grapefruit juice.
- After mixing, give the dose right away. Do not store for future use.
- Keep giving this drug to your child as you have been told by your child’s doctor or other health care provider, even if your child feels well.
What do I do if my child misses a dose?
- Give a missed dose as soon as you think about it.
- If it is close to the time for your child’s next dose, skip the missed dose and go back to your child’s normal time.
- Do not give 2 doses at the same time or extra doses.
How do I store and/or throw out this drug?
All products:
- Store at room temperature in a dry place. Do not store in a bathroom.
- Keep all drugs in a safe place. Keep all drugs out of the reach of children and pets.
- Throw away unused or expired drugs. Do not flush down a toilet or pour down a drain unless you are told to do so. Check with your pharmacist if you have questions about the best way to throw out drugs. There may be drug take-back programs in your area.
Liquid (suspension):
- After opening, throw away any part not used after 1 month.
General drug facts
- If your child’s symptoms or health problems do not get better or if they become worse, call your child’s doctor.
- Do not share your child’s drug with others and do not give anyone else’s drug to your child.
- Some drugs may have another patient information leaflet. If you have any questions about this drug, please talk with your doctor, nurse, pharmacist, or other health care provider.
- If you think there has been an overdose, call your poison control center or get medical care right away. Be ready to tell or show what was taken, how much, and when it happened.
Consumer Information Use and Disclaimer
This generalized information is a limited summary of diagnosis, treatment, and/or medication information. It is not meant to be comprehensive and should be used as a tool to help the user understand and/or assess potential diagnostic and treatment options. It does NOT include all information about conditions, treatments, medications, side effects, or risks that may apply to a specific patient. It is not intended to be medical advice or a substitute for the medical advice, diagnosis, or treatment of a health care provider based on the health care provider’s examination and assessment of a patient’s specific and unique circumstances. Patients must speak with a health care provider for complete information about their health, medical questions, and treatment options, including any risks or benefits regarding use of medications. This information does not endorse any treatments or medications as safe, effective, or approved for treating a specific patient. UpToDate, Inc. and its affiliates disclaim any warranty or liability relating to this information or the use thereof. The use of this information is governed by the Terms of Use, available at https://www.wolterskluwer.com/en/know/clinical-effectiveness-terms.
Last Reviewed Date
2024-06-25
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