Emflaza (deflazacort) is a medication taken once daily to treat a rare, genetic problem called Duchenne muscular dystrophy (DMD). DMD is usually seen in children, and it's known to cause muscle damage and weakness. Emflaza (deflazacort) can be taken by children as young as 2 years old, but this medication can cause a lot of side effects.
Duchenne muscular dystrophy (DMD) in people 2 years and older
Emflaza (deflazacort) is a corticosteroid. It works in your body to lower inflammation and suppress (weaken) your immune system. It's not known exactly how Emflaza (deflazacort) helps people with DMD, but this medication can help improve muscle strength and delay symptoms from getting worse.
Source: DailyMed
Side effects that you should report to your care team as soon as possible:
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
Available in tablets and liquid suspension
Tablets can be crushed if needed
Available in multiple different strengths
Only needs to be taken once a day
Can cause many problems with hormones and growth development
Can be harmful if taken during pregnancy or breastfeeding
Might take a long time to notice an improvement in symptoms
Let your provider know if your child is taking any other medications, including over-the-counter medications and supplements, in case the dose of Emflaza (deflazacort) needs to be adjusted to avoid an interaction.
Emflaza (deflazacort) can be taken with or without food. Don't take Emflaza (deflazacort) with grapefruit juice because this combination can raise medication levels in the body, which can lead to more side effects.
If taking Emflaza (deflazacort) tablets, your child can swallow it whole, or you can crush and mix the tablets with applesauce. Once it's mixed with applesauce, it should be taken right away.
If taking Emflaza (deflazacort) liquid suspension, make sure to shake the bottle well. Only use the measuring device that comes with the suspension to ensure the right dose is given. Don't use household measuring spoons or cups, since this can cause you to measure the wrong dose. Add the suspension to 3 or 4 ounces of juice or milk, mix well, and take it right away.
Throw away any unused Emflaza (deflazacort) liquid suspension after 1 month of opening the bottle.
Don't stop taking Emflaza (deflazacort) suddenly without talking to your provider because it can cause serious problems with hormone levels. If you're taking this medication for more than a few days, your provider will gradually lower the dose until it can be safely stopped.
Let the provider know if you or your child has had a recent infection, currently has an infection, or received a vaccine recently. Depending on your condition, you might have to wait for a period of time before you can safely take Emflaza (deflazacort).
Make sure you're up to date with all of your vaccinations before starting Emflaza (deflazacort). Live vaccines should be given 4 to 6 weeks before starting Emflaza (deflazacort). Speak to your provider if you have questions about vaccines before starting this medication.
Keep away from others who might have chickenpox or measles since catching these can lead to serious reactions while taking Emflaza (deflazacort). Let your provider know right away if you or your child is exposed.
Let your provider know right away if your or your child develops a rash because it could be a sign of a serious allergic reaction.
Your provider will monitor your blood pressure and look for any swelling while you're taking Emflaza (deflazacort). They can advise you on proper diet and supplements if this becomes a problem.
Let your provider know if you or your child experiences any mood or behavior changes while taking Emflaza (deflazacort).
Taking Emflaza (deflazacort) for a long time can have serious side effects, such as a higher risk for bone fractures, growth problems, and vision problems. Discuss with your provider what you should look for if you or your child are on this medication long term.
Emflaza (deflazacort) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Risk factors: Long-term use | Stopping Emflaza (deflazacort) suddenly | Thyroid problems | Hormone problems | Pheochromocytoma | Stress
Emflaza (deflazacort) can change your body's hormone levels, especially if it's taken for a long time. Hormone changes can cause serious and life-threatening problems. If Emflaza (deflazacort) is stopped suddenly, you can have a higher risk of experiencing steroid withdrawal symptoms, which include low appetite, nausea, vomiting, headache, fever, joint pain, and weight loss. If you need to stop taking Emflaza (deflazacort), talk to your provider on how to gradually lower the dose until you can safely stop. Problems with hormone levels can develop for months after stopping Emflaza (deflazacort), so continue to follow up with your provider even after stopping this medication.
Taking Emflaza (deflazacort) for a long time can cause a condition called Cushing's syndrome, which is caused by too much steroid in your body. This can lead to problems like high blood pressure, weight gain, thinning of the arms or legs, purple stretch marks, round face, face swelling, muscle weakness, easy bruising, neck fat, bone problems, acne, menstrual abnormalities, excessive hair growth, depression, or mood swings. Let your provider know if you experience any of these symptoms.
Thyroid problems can affect how Emflaza (deflazacort) gets cleared out of your body. It's recommended to take Emflaza (deflazacort) before starting levothyroxine (Synthroid) treatment for low thyroid levels to lower the risk of serious hormone problems. Let your provider know if you have a history of thyroid problems before taking Emflaza (deflazacort).
Emflaza (deflazacort) can also raise the risk of developing pheochromocytoma crisis in people with pheochromocytoma (tumor in the adrenal glands). Speak to your provider before starting Emflaza (deflazacort) about your risk of developing this life-threatening condition, which leads to low or high blood pressure, high fever, and organ failure.
Risk factors: Diabetes
Emflaza (deflazacort) can raise blood sugar levels, which can make diabetes worse. Emflaza (deflazacort) can make diabetes medications not work as well. Your provider will monitor your blood sugar levels and adjust your diabetes medications if needed while you're taking Emflaza (deflazacort).
Risk factors: High dose | History of or current infection
Steroids like Emflaza (deflazacort) can weaken the immune system and raise your risk of infections. Make sure your immunizations are current and treat all infections before starting this medication. If you or your child develops chickenpox or measles while taking Emflaza (deflazacort), it could be life-threatening. Let your provider know right away if you've been exposed to someone with these illnesses. Emflaza (deflazacort) can raise the risk of reactivation of hepatitis B virus if you had it in the past. Let your provider know right away if you or your child experiences any signs of infection, such as fever, cough, flu-like symptoms, stomach pain, vision problems, or diarrhea.
Risk factors: Heart failure | High blood pressure | Kidney problems | Recent heart attack
Emflaza (deflazacort) can cause damage to the heart and kidneys by raising blood pressure, raising salt and water levels, and causing potassium and calcium loss. Your provider will monitor your blood pressure levels, potassium levels, and signs of swelling and excess body fluid. Let your provider know if you or your child has any heart or kidney problems before starting Emflaza (deflazacort) to make sure this medication is safe to take.
Risk factors: Stomach ulcer | Diverticulitis | Stomach surgery | Ulcerative colitis
Emflaza (deflazacort) can raise the risk of stomach problems, such as getting a tear in the stomach lining. Tell your provider if you or your child has any stomach problems, such as stomach ulcers, infections (e.g., diverticulitis), or recent stomach surgery.
Risk factors: History of mental health conditions | High dose
Emflaza (deflazacort) might cause changes in your mood or behavior, which can happen within a few days or weeks of starting this medication. Let your provider know right away if you notice any mood swings (feeling extreme excitement, happiness, or depression), hallucinations (hearing or seeing things that aren't there), trouble sleeping (insomnia), or a loss of contact with reality while taking this medication.
Risk factors: Children and adolescents
Emflaza (deflazacort) can reduce bone formation and lead to bone loss. This can later raise your risk for bone fractures. In some cases, Emflaza (deflazacort) can cause blood supply problems to your bones. Your provider will monitor your or your child's bones, growth, and development while taking this medication. Speak to your provider if you have concerns.
Risk factors: Cataracts | Glaucoma
Emflaza (deflazacort) might raise your risk of eye problems, such as cataracts, glaucoma, and eye infections. Let your provider know if you or your child develops blurry vision or other vision problems while taking Emflaza (deflazacort).
Although rare, some people experience serious skin rashes while taking Emflaza (deflazacort). There have been reports of a life-threatening condition called toxic epidermal necrolysis (TEN), which causes blistering and peeling of the skin, that can occur within 8 weeks of starting Emflaza (deflazacort). Let your provider know right away at the first sign of a rash, and they can advise you how to safely stop this medication if needed.
Risk factors: Long-term use
Although rare, there have been reports of people developing a type of cancer called Kaposi's sarcoma when taking corticosteroids like Emflaza (deflazacort) for a long time. Discuss the risk with your provider if you're to take this medication long term.
Risk factors: History of nerve conditions | Taking neuromuscular blocking medications
Emflaza (deflazacort) can raise the risk of serious muscle weakness that can even sometimes affect breathing muscles in people with a history of nerve conditions (e.g., myasthenia gravis) or who are taking neuromuscular blocking medications (e.g., pancuronium). Recovery can take weeks to years after stopping Emflaza (deflazacort). Let your provider know if you experience muscle weakness, breathing problems, or vision problems while taking this medication.
Emflaza (deflazacort) can cause birth defects and restrict the growth of unborn babies if given during pregnancy. It can also pass into breast milk and cause side effects in breastfed babies. Speak with your provider about the risks and benefits to you and your baby before taking Emflaza (deflazacort) during pregnancy or breastfeeding.
Emflaza (deflazacort) shouldn't be used in children under 2 years old because it hasn't been proven to be safe or work well in this age group. In addition, the liquid suspension contains a preservative called benzyl alcohol. Infants who take benzyl alcohol can have serious reactions like "gasping syndrome", where they have trouble breathing, buildup of acid in the body (metabolic acidosis), and gasp for air. If your child has these symptoms, seek medical help right away.
Risk factors: High dose | History of blood clotting problems
Although rare, Emflaza (deflazacort) might raise your risk of getting blood clots. If you have a history of getting blood clots, speak with your provider before starting Emflaza (deflazacort) to see if this medication is safe to take.
Dosing will depend on your or your child’s weight. The typical dose is 0.9 mg/kg by mouth once daily.
Duchenne muscular dystrophy (DMD) in people 2 years and older
Allergies or allergic reactions
Conditions affecting certain hormones (e.g., low adrenal hormones)
Eye conditions
Lung conditions
Gut conditions (e.g., ulcerative colitis)
Conditions related to blood cells
Certain cancers (e.g., leukemia
Nervous system conditions (e.g., multiple sclerosis)
Joint conditions (e.g., rheumatoid arthritis)
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