Emflaza Side Effects: What to Expect & How to Manage Them

Key Takeaways:

• Emflaza (deflazacort) is the only FDA-approved corticosteroid for treating Duchenne muscular dystrophy (DMD) in patients 2 years and older
• Common side effects include facial puffiness (Cushingoid appearance), weight gain, increased appetite, and upper respiratory infections
• Serious side effects requiring monitoring include bone health issues, cataracts, growth delays, adrenal insufficiency, and behavioral changes
• Emflaza can help delay loss of walking ability by 1.4 to 2.5 years and improve muscle strength and pulmonary function
• Never stop Emflaza suddenly—gradual dose reduction under medical supervision is required to prevent dangerous adrenal crisis
• PTC Cares patient assistance programs can help eliminate or minimize out-of-pocket costs for eligible patients

Emflaza (deflazacort) represents a significant advancement in treating Duchenne muscular dystrophy (DMD), offering hope to boys and families affected by this progressive genetic disorder. As the only FDA-approved corticosteroid specifically indicated for DMD, Emflaza has been shown to improve muscle strength, delay loss of walking ability, and potentially slow disease progression. While this medication can provide substantial benefits, understanding its potential side effects is essential for successful long-term treatment. This comprehensive guide will help you know what to expect, when to call your doctor, how to manage side effects, and how to access copay assistance programs to make treatment affordable for your family.

What Is Emflaza (Deflazacort) and How Does It Work?

Emflaza (deflazacort) is a corticosteroid—specifically a glucocorticoid—that works as an anti-inflammatory and immunosuppressive agent. According to the U.S. Food and Drug Administration, Emflaza was approved in 2017 as the first corticosteroid with an FDA indication specifically for Duchenne muscular dystrophy.

Deflazacort is converted by the body into an active metabolite called 21-desDFZ, which helps decrease chronic inflammation and suppress immune system activity that contributes to muscle damage in DMD. While Emflaza is not a cure for muscular dystrophy, it may improve muscle strength and help slow the progression of the disease.

Understanding Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is a severe, progressive genetic disorder that primarily affects boys. DMD is caused by mutations in the dystrophin gene, which results in low levels or absence of dystrophin—a protein that helps keep muscle cells intact as they stretch and contract with motion. Without adequate dystrophin, muscle cells become damaged with each contraction and eventually die, leading to progressive muscle weakness and wasting.

FDA-Approved Use

Emflaza is approved to treat Duchenne muscular dystrophy in patients 2 years of age and older. The medication is taken orally once daily, with dosing based on the patient’s weight (approximately 0.9 mg/kg/day).

How Emflaza Can Be Taken

Emflaza is available in two forms:

• Tablets: Available in 6mg, 18mg, 30mg, and 36mg strengths. Tablets can be swallowed whole or crushed and mixed with applesauce for immediate consumption
• Oral suspension: 22.75 mg/mL liquid formulation that should be shaken well before use

Emflaza can be taken with or without food, but should NOT be taken with grapefruit juice, as this can increase medication levels in the body and lead to more side effects.

Common Emflaza Side Effects: What Most Patients Experience

Understanding which side effects are most common helps families prepare for treatment. The Mayo Clinic notes that while many patients taking Emflaza experience at least one side effect, the severity varies significantly from person to person.

Physical Appearance Changes

Facial Puffiness or Cushingoid Appearance (Common):

One of the most frequently reported side effects is the development of a Cushingoid appearance, which includes facial puffiness (moon face), increased fat around the midsection and upper back (buffalo hump), and a rounder facial shape. This occurs because corticosteroids like Emflaza affect how the body stores fat.

What to expect:

• Gradual rounding of the face over weeks to months
• Increased fat deposits in the midsection, upper back, or neck
• Pink or purple stretch marks may develop on the skin
• Thinning, fragile skin that bruises easily

Management tips:

• These changes are expected with corticosteroid treatment and do not mean the medication isn’t working
• Maintain a healthy diet focused on whole foods, lean proteins, and vegetables
• Limit sodium intake to reduce fluid retention
• Stay physically active within your child’s capabilities
• Remember that appearance changes may reverse when treatment is eventually discontinued under medical supervision
• Seek emotional support for your child if appearance changes affect self-esteem

Weight and Appetite Changes

Weight Gain and Increased Appetite (Very Common):

Weight gain is one of the most common side effects of Emflaza treatment. According to clinical studies, patients taking deflazacort experience less weight gain compared to those taking prednisone, though weight gain still occurs.

Management tips:

• Focus on nutrient-dense foods rather than calorie-dense foods
• Offer smaller, more frequent meals to help manage increased appetite
• Encourage physical activity appropriate for your child’s abilities
• Limit sugary snacks and beverages
• Work with a dietitian who has experience with DMD patients on corticosteroids
• Monitor weight regularly and report significant changes to your healthcare team
• Understand that some weight gain may help boys with DMD maintain strength

Unwanted Hair Growth (Hirsutism):

Some patients experience increased hair growth on the face, arms, or back.

Management tips:

• Gentle hair removal methods if desired and age-appropriate
• Consult with your healthcare provider about safe hair removal options
• Remember that this is a common corticosteroid side effect

Respiratory and Infection-Related Side Effects

Upper Respiratory Tract Infections and Colds (Very Common):

Because Emflaza suppresses the immune system, patients are at increased risk for infections. Upper respiratory tract infections, coughs, and common colds are frequently reported.

Management tips:

• Practice good hand hygiene—frequent handwashing is essential
• Avoid close contact with people who are sick
• Ensure your child is up-to-date on all recommended vaccinations before starting Emflaza
• Contact your healthcare provider promptly if your child develops fever, persistent cough, or other signs of infection
• Avoid crowded places during cold and flu season when possible
• Report any wounds that don’t heal properly

Cough and Nasopharyngitis:

Coughing and inflammation of the nose and throat are common, especially in the first months of treatment.

Management tips:

• Use a humidifier to keep air moist
• Encourage adequate hydration
• Contact your doctor if cough persists or worsens
• Watch for signs of more serious respiratory infections

Urinary Changes

Frequent Daytime Urination (Common):

Increased frequency of urination during the day is a commonly reported side effect.

Management tips:

• Ensure easy bathroom access throughout the day
• Communicate with school staff about potential bathroom needs
• Stay hydrated despite increased urination
• Report painful urination or other urinary symptoms immediately

Serious Emflaza Side Effects Requiring Medical Attention

While many side effects are manageable, Emflaza carries several serious risks that require careful monitoring. The FDA requires specific warnings about these potentially severe complications.

Alterations in Endocrine Function and Adrenal Insufficiency

CRITICAL WARNING: Adrenal Insufficiency and Withdrawal Syndrome

This is one of the most serious risks of Emflaza treatment. Corticosteroids like Emflaza can suppress your body’s natural production of cortisol. If Emflaza is stopped suddenly, acute adrenal insufficiency can occur, which can be FATAL.

Why this is critical: When you take corticosteroids for more than a few days, your adrenal glands stop producing natural cortisol. Your body needs time to restart this production when stopping the medication.

Warning signs of adrenal insufficiency:

• Severe nausea and vomiting
• Loss of appetite
• Unusual weakness or fatigue that is more severe than typical DMD fatigue
• Dizziness or fainting
• Abdominal pain
• Confusion or coma
• Dehydration
• High fever
• Low blood pressure (hypotension)

What you must do:

• NEVER stop Emflaza suddenly without your doctor’s guidance
• NEVER skip doses for more than 24 hours
• Inform all healthcare providers that your child is taking Emflaza
• During times of stress (illness, surgery, injury), your child may need supplemental “stress doses” of corticosteroids
• Always follow your doctor’s tapering schedule if discontinuing treatment
• Seek emergency care immediately if you suspect adrenal crisis

Bone Health and Osteoporosis

Reduced Bone Density and Increased Fracture Risk:

Long-term corticosteroid use can lead to decreased bone mineral density, increasing the risk of fractures. According to clinical research, deflazacort may affect bone health, with vertebral and long bone fractures being a concern with prolonged use.

Why this happens: Corticosteroids interfere with calcium absorption and bone formation while increasing bone breakdown.

Management and monitoring:

• Regular bone density scans (DXA scans) to monitor bone health
• Calcium supplementation: 1,200 mg per day recommended for long-term corticosteroid use
• Vitamin D supplementation: 800 IU per day (your doctor may check vitamin D levels and adjust dosing)
• Weight-bearing activities as tolerated to promote bone strength
• Your doctor may prescribe bisphosphonates if bone density decreases significantly
• Report any bone pain, back pain, or suspected fractures immediately
• Be aware that boys with DMD often have reduced bone density even before starting steroids

Eye Problems: Cataracts and Glaucoma

Cataract Formation and Increased Intraocular Pressure:

According to clinical studies comparing deflazacort with prednisone, deflazacort is associated with a greater risk of developing cataracts. Most cataracts that develop are asymptomatic (not vision-impairing) initially, but monitoring is essential.

What to watch for:

• Cloudy or blurry vision
• Increased sensitivity to glare or light
• Difficulty seeing at night
• Seeing halos around lights
• Fading or yellowing of colors

Required monitoring:

• Regular ophthalmologic examinations to monitor for cataracts and glaucoma
• If corticosteroid treatment continues for more than 6 weeks, intraocular pressure should be monitored
• Report any vision changes to your healthcare provider immediately
• Most cataracts that develop are monitored and may not require immediate treatment

Growth and Development Effects

Growth Delay and Height Suppression:

Long-term use of corticosteroids can slow growth and development in children. Clinical studies show that deflazacort may be associated with growth retardation.

What your healthcare team will do:

• Regular height and growth measurements at each visit
• Growth charts to track growth velocity
• Consideration of the balance between delaying disease progression and growth effects
• Possible dose adjustments if growth delay is significant

Important to understand: The benefits of Emflaza in delaying loss of walking ability and maintaining strength must be balanced against potential effects on growth. Your healthcare team will work with your family to find the optimal approach.

Blood Sugar Changes (Hyperglycemia)

High Blood Sugar (Hyperglycemia):

Emflaza can increase blood sugar levels. Symptoms of high blood sugar include increased thirst, increased urination, unusual weakness or fatigue, and blurry vision.

Management tips:

• Your healthcare provider will monitor blood sugar levels regularly
• Limit sugary foods and beverages
• Report symptoms of increased thirst, frequent urination, or unusual fatigue
• In rare cases, diabetes medications may be needed

Behavioral and Mood Changes

Behavioral Disorders and Mood Changes:

Emflaza can cause behavioral and mood changes ranging from mild irritability to more serious psychological effects. Studies comparing deflazacort with prednisone found that deflazacort was associated with less abnormal behavior than prednisone.

Symptoms to watch for:

• Restlessness or irritability
• Mood swings
• Anxiety
• Sleep disturbances
• Changes in personality
• In rare cases: euphoria, psychotic reactions, depression

What to do:

• Report any behavioral or mood changes to your healthcare provider promptly
• Keep a journal of behavioral patterns to share with your doctor
• Your doctor may adjust the dose or recommend supportive treatments
• Seek immediate medical attention if your child experiences severe mood changes, depression, or thoughts of self-harm

Increased Risk of Infections

Serious and Potentially Fatal Infections:

Because Emflaza suppresses the immune system, there is an increased risk of infections, including serious infections that can potentially be fatal.

Critical precautions:

• Warn your child to avoid exposure to chickenpox or measles
• Alert your healthcare provider IMMEDIATELY if your child is exposed to chickenpox or measles
• Seek medical attention immediately if your child develops fever, chills, persistent cough, sore throat, or other signs of infection
• Report wounds that don’t heal properly
• Pain or trouble when urinating may indicate infection

Cardiovascular Effects

High Blood Pressure and Fluid Retention:

Emflaza can cause increased blood pressure and water retention.

Management:

• Regular blood pressure monitoring
• Dietary salt restriction if recommended by your doctor
• Potassium supplementation may be needed
• Report swelling in hands, feet, or face

Gastrointestinal Effects

Risk of Gastrointestinal Perforation:

There is an increased risk of developing a hole (perforation) in the stomach or intestines in patients with certain stomach or intestinal disorders when taking corticosteroids like Emflaza.

Warning signs:

• Severe abdominal pain
• Fever with abdominal pain
• Nausea and vomiting
• Sudden worsening of symptoms

Action required: Seek emergency medical care if your child experiences severe abdominal pain.

Serious Skin Reactions

Toxic Epidermal Necrolysis and Severe Rashes:

In rare cases, deflazacort has been associated with serious skin reactions including toxic epidermal necrolysis.

What to do:

• Discontinue Emflaza at the first sign of rash unless the rash is clearly not drug-related
• Seek medical attention immediately for any rash that:
• Spreads rapidly
• Blisters or peels
• Is accompanied by fever
• Involves the mouth, eyes, or genital area

Allergic Reactions

Hypersensitivity and Anaphylaxis:

Rare instances of anaphylaxis (severe allergic reactions) have occurred in patients receiving corticosteroid therapy, including Emflaza.

Signs of severe allergic reaction:

• Skin rash, itching, or hives
• Swelling of the face, lips, tongue, or throat
• Difficulty breathing
• Chest tightness

Action required: Seek emergency medical care immediately if these symptoms occur.

Important Vaccination Considerations

Immunizations must be carefully managed when your child is taking Emflaza.

Before Starting Emflaza

• Immunizations should be up-to-date according to immunization guidelines BEFORE starting Emflaza
• Live-attenuated or live vaccines should be administered at least 4 to 6 weeks BEFORE starting Emflaza
• Examples of live vaccines include measles, mumps, rubella (MMR), varicella (chickenpox), live flu vaccine (nasal), rotavirus, and some others

During Emflaza Treatment

• Do NOT administer live or live-attenuated vaccines to patients receiving immunosuppressive doses of corticosteroids
• Inactivated vaccines can generally be given, but the immune response may be reduced
• Your child should not be around other household members who receive live virus vaccines, as there is a chance they could pass the virus to your child

Always discuss vaccination plans with your healthcare provider.

When to Call Your Doctor Immediately

Seek medical attention right away if you experience:

• Signs of adrenal insufficiency: severe weakness, nausea, vomiting, dizziness, especially after missing doses
• Severe allergic reactions: difficulty breathing, swelling of face/lips/tongue/throat, hives
• Signs of serious infection: high fever, chills, persistent cough, wounds that don’t heal
• Exposure to chickenpox or measles
• Severe abdominal pain: could indicate gastrointestinal perforation
• Vision changes: blurry vision, halos, difficulty seeing
• Severe skin rash: especially if spreading, blistering, or accompanied by fever
• Severe behavioral or mood changes: including thoughts of self-harm
• Signs of low potassium: muscle pain or cramps, unusual weakness, fast or irregular heartbeat
• Any symptom that feels severe or life-threatening

How to Manage Side Effects Successfully

Open Communication is Essential

Maintain regular communication with your child’s healthcare team. Report any side effects at follow-up appointments, even if they seem minor. Your doctor can adjust the treatment plan or provide medications to manage symptoms.

Keep a Side Effect Journal

Track when side effects occur, their severity, what helps, and any patterns you notice. This information helps your doctor make informed treatment decisions. Include:

• Date and time symptoms occur
• Severity (mild, moderate, severe)
• What makes symptoms better or worse
• Impact on daily activities
• Current dose of Emflaza

Attend All Monitoring Appointments

Regular check-ups and monitoring are essential for catching potential problems early. Required monitoring typically includes:

• Regular visits to measure growth and weight
• Bone density scans (DXA scans)
• Ophthalmologic examinations for cataracts and glaucoma
• Blood pressure monitoring
• Blood tests to check electrolytes, blood sugar, and other parameters
• Assessment of muscle strength and function

Important Medication Interactions

Tell your healthcare provider about ALL medications your child takes, including:

• Moderate or strong CYP3A4 inhibitors (like clarithromycin, certain antifungals): These can increase deflazacort levels. Your doctor may need to reduce Emflaza dose to one-third of the usual dose
• Moderate or strong CYP3A4 inducers (like certain seizure medications): These can decrease deflazacort levels and should generally be avoided
• Insulin or other diabetes medications
• NSAIDs like aspirin or ibuprofen
• Over-the-counter medications, vitamins, and supplements
• Herbal products

Important: Do NOT give your child grapefruit or grapefruit juice while taking Emflaza, as this can increase medication levels and side effects.

Special Considerations During Times of Stress

During times of physical stress (illness, surgery, injury), your child may need increased doses of corticosteroids. Always inform healthcare providers about Emflaza use before any medical procedure.

Living Well with Emflaza: Lifestyle and Support Tips

Nutritional Support

Proper nutrition is crucial for managing side effects:

• Calcium and Vitamin D: Essential for bone health. Consider 1,200 mg calcium and 800 IU vitamin D daily (confirm with your doctor)
• Low sodium diet: Helps reduce fluid retention and blood pressure
• Balanced meals: Focus on lean proteins, whole grains, fruits, and vegetables
• Limit simple sugars: Helps manage weight and blood sugar
• Adequate hydration: Important for overall health
• Consider working with a registered dietitian experienced in DMD and corticosteroid management

Physical Activity

Maintain appropriate physical activity based on your child’s abilities:

• Weight-bearing activities help maintain bone density
• Physical therapy to maintain strength and function
• Occupational therapy to maintain independence in daily activities
• Balance activity with rest

Emotional and Psychological Support

Living with DMD and managing treatment side effects can be emotionally challenging:

• Consider counseling or support groups for your child
• Family counseling can help the whole family cope
• Connect with other DMD families through organizations like Parent Project Muscular Dystrophy
• Address appearance-related concerns with compassion and support
• Watch for signs of depression or anxiety and seek help promptly

School and Daily Life

• Communicate with school staff about your child’s needs (bathroom access, infection precautions)
• Have a plan for administering medication consistently at the same time each day
• Keep an emergency plan in place for adrenal crisis
• Consider a medical alert bracelet indicating corticosteroid use

Understanding the Risk vs. Benefit of Emflaza

While this guide covers many potential side effects, it’s important to remember that:

• Not all patients experience all side effects
• The severity of side effects varies widely from person to person
• Many side effects can be managed with proper monitoring and treatment
• According to research from the American Academy of Neurology, the benefits of corticosteroid treatment in DMD are substantial, including delaying loss of ambulation by 1.4 to 2.5 years, improving pulmonary function, reducing need for scoliosis surgery, and potentially improving survival
• Emflaza may be associated with less weight gain and fewer behavioral changes compared to prednisone, though it may have greater effects on bone health and cataracts
• Your healthcare team can help you manage side effects while maximizing benefits

The decision to use Emflaza involves carefully weighing the demonstrated benefits of prolonging walking ability and maintaining strength against the potential side effects. For many families, the benefits of gaining additional years of mobility justify corticosteroid use when side effects are proactively managed.

Affording Your Emflaza Treatment: PTC Cares Patient Assistance

One concern that shouldn’t add to your family’s burden is the cost of treatment. Emflaza can be expensive, with some patients facing high out-of-pocket costs even with insurance. Fortunately, the manufacturer offers comprehensive patient assistance programs designed to eliminate or minimize costs for eligible patients.

PTC Cares Patient Support Program

PTC Therapeutics, the manufacturer of Emflaza, offers PTC Cares—a dedicated patient support program that helps families access Emflaza and navigate the financial aspects of treatment. According to information from PTC Therapeutics, the program provides comprehensive support including:

• Dedicated Case Managers who guide families through every step
• Insurance verification and benefits investigation
• Assistance with prior authorization requirements
• Financial assistance programs tailored to your insurance type
• Coordination with specialty pharmacy for medication delivery

Financial Assistance Based on Insurance Type

For Private or Commercial Insurance:

PTC is committed to eliminating patient out-of-pocket expenses for commercially insured patients. Your PTC Cares Case Manager will automatically enroll you into programs that eliminate your cost in most cases. Some state regulations may require a small monthly payment (such as $10), but for most commercially insured patients, costs can be completely eliminated.

For Public or Government Insurance (Medicaid/Medicare):

If your insurance is provided by the government, you may have certain out-of-pocket costs. Your PTC Cares Case Manager will refer you to third-party patient foundations that can provide financial assistance based on your eligibility. For most people on Medicaid, prescription drug copays range from $4 to $9 per month, and some states eliminate the copay requirement altogether.

For Uninsured Patients:

If you do not have insurance coverage, your PTC Cares Case Manager will offer Emflaza free of charge if you meet eligibility criteria through the patient assistance program.

Bridge Program for Treatment Continuity

PTC offers a Bridge Program to ensure that patients can continue treatment while waiting for insurance verification of coverage. Under this program, eligible patients receive Emflaza free of charge during the approval process, ensuring no interruption in treatment.

How to Get Started with PTC Cares

Getting started is simple:

1. Complete the Prescription Start Form: Work with your child’s healthcare provider to complete and sign the form
2. Submit to PTC Cares: The form can be faxed or submitted electronically
3. Get Assigned a Case Manager: You’ll be assigned a dedicated Case Manager who will guide you through the entire process
4. Receive Support: Your Case Manager will verify insurance, determine eligibility for assistance programs, and coordinate medication delivery through the specialty pharmacy

How QuickRx Specialty Pharmacy Can Help

At QuickRx Specialty Pharmacy, we understand that managing Duchenne muscular dystrophy is challenging enough without worrying about medication costs. While PTC Cares provides comprehensive support directly from the manufacturer, our team can also assist families in navigating patient assistance programs for Emflaza and other specialty medications your child may need.

Our services include:

• Guidance on accessing manufacturer patient assistance programs
• Help understanding your insurance benefits
• Support with prior authorization requirements
• Coordination with your healthcare team
• Assistance with other specialty medications for DMD and related conditions

Don’t let medication costs prevent your child from getting the treatment they need. Contact QuickRx Specialty Pharmacy at (917) 839-2525 or (800) 496-6111 to learn more about accessing patient assistance for Emflaza and other specialty medications.

We also help patients access copay assistance for many other specialty medications. Visit our copay assistance page to see how we can help with your family’s specific medication needs.

Questions to Ask Your Doctor About Emflaza

Come prepared to your appointments with questions like:

• At what age should my child start Emflaza treatment?
• What is the appropriate dose for my child’s weight?
• What side effects are most likely given my child’s specific situation?
• How will you monitor my child for serious side effects?
• What symptoms require immediate medical attention?
• How often will my child need bone density scans and eye exams?
• Should we see a dietitian to help manage weight and bone health?
• What should I do if my child misses a dose?
• What is the plan if we need to discontinue Emflaza in the future?
• How do I handle sick days or times of stress when my child may need extra steroids?
• Can my child receive vaccinations while on Emflaza?
• What other medications should be avoided while taking Emflaza?
• How do Emflaza’s side effects compare to prednisone?
• Am I eligible for the PTC Cares patient assistance program?
• What are the long-term expectations for treatment duration?

Additional Resources for Duchenne Muscular Dystrophy and Emflaza

For more information about Emflaza, Duchenne muscular dystrophy, and support resources:

• Parent Project Muscular Dystrophy – Leading organization providing support, resources, and advocacy for DMD families
• Muscular Dystrophy Association (MDA) – Comprehensive resources and support for families affected by muscular dystrophy
• CureDuchenne – Research and resources focused on finding a cure for DMD
• Emflaza Official Website – Information directly from the manufacturer
• American Academy of Neurology – Clinical guidelines for DMD treatment
• National Organization for Rare Disorders (NORD) – Information about rare diseases including DMD

Frequently Asked Questions About Emflaza Side Effects

How long does it take to see benefits from Emflaza?

According to clinical studies, improvements in muscle strength can be seen within six months to two years of starting Emflaza treatment. The full benefit of delaying loss of walking ability may take longer to become apparent. Your child’s neurologist will monitor muscle strength and motor function regularly to assess treatment response.

Will my child definitely get all these side effects?

No. Not every patient experiences all side effects listed. Some side effects like facial puffiness and weight gain are very common, while others like cataracts and growth delay develop over longer periods of time. The type and severity of side effects vary significantly from person to person. Regular monitoring helps detect side effects early so they can be managed appropriately.

Can Emflaza side effects be prevented?

Some side effects can be minimized through proactive management. For example, calcium and vitamin D supplementation can help protect bone health, dietary modifications can help manage weight gain, and regular eye exams can detect cataracts early. However, some side effects like Cushingoid appearance are expected effects of corticosteroid treatment and cannot be completely prevented.

How does Emflaza compare to prednisone in terms of side effects?

According to clinical research, deflazacort (Emflaza) and prednisone have somewhat different side effect profiles. Studies show that deflazacort is associated with less weight gain and fewer behavioral problems compared to prednisone. However, deflazacort appears to have greater effects on bone health, cataracts, and growth parameters. Your healthcare provider can help determine which medication is most appropriate for your child based on individual factors.

What happens if we have to stop Emflaza?

Emflaza should NEVER be stopped suddenly. If treatment needs to be discontinued, your doctor will create a tapering schedule to gradually reduce the dose over time. This allows your child’s adrenal glands to resume natural cortisol production safely. The tapering schedule depends on how long your child has been taking Emflaza and the dose. Always follow your doctor’s tapering instructions carefully.

How long will my child need to take Emflaza?

Most boys with DMD take corticosteroids long-term, often for many years. The duration depends on individual response to treatment, tolerance of side effects, and disease progression. Some patients continue corticosteroids throughout their ambulatory years and beyond. Your healthcare team will regularly assess whether the benefits continue to outweigh the side effects.

What should I do if my child vomits after taking Emflaza?

If your child vomits shortly after taking Emflaza (within 30 minutes), contact your healthcare provider or pharmacist for guidance on whether to give another dose. If vomiting occurs more than 30 minutes after the dose, do not give another dose. Never double the next dose to make up for vomiting. If vomiting persists, contact your doctor as this could be a sign of illness requiring stress dose steroids.

Can my child participate in sports while taking Emflaza?

Physical activity is generally encouraged for children with DMD taking Emflaza, as it helps maintain muscle strength and bone health. However, because Emflaza can affect bone density, your child may be at increased risk for fractures. Activities should be appropriate for your child’s abilities and safety. Discuss specific activities with your healthcare team, physical therapist, and consider the stage of your child’s disease progression.

Will the Cushingoid appearance go away?

Physical appearance changes like facial puffiness and increased fat deposits are common with long-term corticosteroid use. These changes may improve somewhat over time as the body adjusts, but they typically persist as long as treatment continues. If Emflaza is eventually discontinued under medical supervision, these appearance changes will gradually reverse, though this process takes time.

How do I know if my child’s growth is being affected?

Your healthcare provider will measure your child’s height at each visit and plot growth on standard growth charts. They will track growth velocity (how quickly your child is growing over time) to determine if growth delay is occurring. If significant growth suppression occurs, your doctor may discuss options such as dose adjustment or weekend-only dosing regimens, balanced against the benefits of treatment.

What if my child is exposed to chickenpox or measles?

This is a medical urgency. Contact your healthcare provider IMMEDIATELY if your child is exposed to chickenpox or measles. Your doctor may prescribe preventive treatment such as varicella-zoster immune globulin (VZIG) for chickenpox exposure. Because Emflaza suppresses the immune system, these infections can be severe or even fatal in patients taking corticosteroids.

Can Emflaza affect my child’s behavior at school?

Behavioral changes including restlessness, irritability, and mood swings can occur with corticosteroid treatment. According to studies, deflazacort is associated with fewer behavioral problems compared to prednisone. If you notice behavioral changes affecting school performance or social interactions, discuss this with your healthcare provider. Dose adjustments or supportive interventions may help. Communicate with school staff about potential behavioral effects so they can provide appropriate support.

Is there a generic version of Emflaza available?

While a generic version of deflazacort has been FDA-approved, Emflaza is currently the brand name product marketed in the United States. The availability and cost of generic deflazacort may vary. Discuss with your healthcare provider and insurance company about which formulation is covered and most appropriate for your child. Some prescriptions may specify “Dispense as Written” (DAW) to ensure the brand name Emflaza is dispensed.

How much does Emflaza cost without assistance?

Emflaza is an expensive specialty medication. Without insurance or assistance programs, costs can range from several thousand to over $10,000 per month depending on the dose. However, most families do not pay anywhere near these amounts due to insurance coverage and patient assistance programs. The PTC Cares program is specifically designed to eliminate or minimize out-of-pocket costs for eligible patients. Contact PTC Cares at 1-844-EMFLAZA to learn about your specific financial assistance options.

Can my child take other medications while on Emflaza?

Many medications can be taken with Emflaza, but some require dose adjustments or should be avoided. Certain medications (moderate or strong CYP3A4 inhibitors like clarithromycin) can increase Emflaza levels and may require reducing the Emflaza dose. Other medications (CYP3A4 inducers) can decrease Emflaza effectiveness. Always inform ALL healthcare providers that your child is taking Emflaza, and check with your doctor or pharmacist before starting any new medications, including over-the-counter products and supplements.

What is the Benzyl Alcohol warning for the oral suspension?

Emflaza oral suspension contains benzyl alcohol (10.45 mg per mL), which may cause seizures and other serious side effects in premature or low birth-weight infants. Emflaza is only approved for use in children 2 years and older, so this is primarily a concern if the suspension is accidentally given to infants under 2 years old. The oral tablets do not contain benzyl alcohol. For children 2 years and older who can swallow tablets or have them crushed in applesauce, tablets may be preferred to avoid benzyl alcohol exposure.

Conclusion: Managing Side Effects for Successful Treatment

Understanding Emflaza side effects empowers families to take an active role in treatment management. While side effects can be concerning, most are manageable with proper monitoring, proactive interventions, and open communication with your healthcare team. The key to successful long-term treatment is staying informed, reporting symptoms promptly, attending all monitoring appointments, and working closely with your medical team.

Remember that Emflaza offers substantial benefits for boys with Duchenne muscular dystrophy, including delaying loss of walking ability, improving muscle strength and pulmonary function, and potentially improving long-term outcomes. The decision to use Emflaza involves carefully balancing these benefits against potential side effects, with the understanding that many side effects can be effectively managed.

Most importantly, never stop Emflaza suddenly or skip doses without medical guidance. Acute adrenal insufficiency can be life-threatening, so always follow your doctor’s instructions for dosing and tapering.

Need help managing your Emflaza treatment costs? Contact QuickRx Specialty Pharmacy today at (917) 839-2525 or (800) 496-6111 for assistance with patient assistance programs and copay assistance for Emflaza and other specialty medications your family may need.

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Last Updated: November 5, 2025
Reviewed By: QuickRx Clinical Team
Medical Disclaimer: Information compiled from FDA prescribing information, peer-reviewed clinical studies, and authoritative medical sources