Learn how to talk to your son's healthcare provider about staying on EMFLAZA.



The following story is of an individual situation and may not be the typical experience for all patients. Consult your healthcare professional about the best treatment plan for you or your child. Participants are being paid by PTC therapeutics and are not medical professionals.

A Real-World Story







EMFLAZA® (deflazacort) is a prescription medicine used to treat Duchenne muscular dystrophy (DMD) in patients 2 years of age and older.

Do not use if you have hypersensitivity, including allergic reactions, to deflazacort or any inactive ingredients.

Duchenne muscular dystrophy affects 1 in 3,600-6,000 live male births.

Manny was one of them.

Manny: My name is Manny and I live in Ossining for seven years and I'm 15.

Lydia: My name is Lydia Aviles. I'm originally from Puerto Rico. My family live in Isabela, in the northwest side.

Manny: There's tasty food.

Lydia: And what else? What do you like practice there?

Manny: Surfing?

Lydia: Surfing.

Manny: There's a lot of mosquitos.

Lydia: [laughs]

There are two corticosteroids used to treat boys with Duchenne muscular dystrophy and they are not the same.

Lydia: So, I remember Manny start to use the medication almost at 11. When Manny start with the prednisone at this rate, the body start to change. Because Manny was very skinny. When he used the prednisone, Manny had won a lot of weight. So that is the first part I'm scared or concerned. He looked in the mirror he said, “Why do I look in that way? I don't want to continue with this medication because maybe in the future, affect some part of my body.”

Lydia: And then after that, I meet the new doctor in New York. It's the MDA clinic. And he's a very good doctor. Excellent. The doctor continued the treatment, but he said–I remember he said, “I'm waiting for one medication, the name is EMFLAZA.” So, I say, “Oh!” Something showed me hope in this patch because it's very hard. And then Manny is start to look information about EMFLAZA. And then when he sees the doctor on the next visit he say, “Are you asking for my medication? When I start with EMFLAZA?” So, the doctor say, “Soon, Manny.”

In time, Manny and his mom were able to work with the doctor to switch to EMFLAZA.

Lydia: The doctor always—he's in contact with us. He called me say, “You know what, I changed your scheduling for the appointment because I need to talk to you.” When I go there, he say, “Okay I'm gonna start the process with the EMFLAZA medication.”

EMFLAZA is the first and only FDA-approved corticosteroid designed to help maintain muscle strength and function.

EMFLAZA is the only corticosteroid approved by the FDA for the treatment of DMD in boys 2 years of age and older.

Lydia: And then he said, “We are lucky because I fighting five times for this medication.” When he said that I feel so, “Yes! He did it!” And then few days after that someone call me and say, “I call you because I want to schedule the delivery for the medication.” And then I say, “Wow, it's real!” Before he used EMFLAZA I remember Manny has, you know, a lot of change. One of the weight, he won more weight. And then when he start to use EMFLAZA just start to change a lot. A lot of changes. Very different than the medication before.

Weight gain can be tough for any boy, but added weight to deteriorating muscles can be even harder to deal with.

EMFLAZA may be able to help.

Boys taking deflazacort experienced less weight gain compared to those taking prednisone

Deflazacort: 27.9% (n=19/68);

prednisone: 34.9% (n=22/63)

Based on clinical study results. Individual weight changes may vary.

Lydia: He say, “Mommy, I feel good with this medication. I think I'm lucky.” “When I look in the mirror, I see a lot of, you know, a lot of changes in my life. Right now, I feel more confidence.” Manny is a big gift for me. He showed me to be more strong. I never give up. I feel very blessing because Manny has a good spirit. He's strong like me. When I saw him, I saw me in him. So it's something magical.

Learn more at EMFLAZA.com


What is EMFLAZA® (deflazacort) used for?

EMFLAZA is a prescription medicine used to treat Duchenne muscular dystrophy (DMD) in patients 2 years of age and older.

When should I not take EMFLAZA?

Do not use if you have had hypersensitivity, including allergic reactions, to deflazacort or any of the inactive ingredients.

What warnings should I know about EMFLAZA?

  • EMFLAZA can cause changes in endocrine function. Do not stop taking EMFLAZA, or change the amount you are taking, without first checking with your healthcare provider, as there may be a need for gradual dose reduction to decrease the risk of adrenal insufficiency and steroid “withdrawal syndrome”. Acute adrenal insufficiency can occur if corticosteroids are withdrawn abruptly, and can be fatal. A steroid “withdrawal syndrome,” seemingly unrelated to adrenocortical insufficiency, may also occur following abrupt discontinuance of corticosteroids. For patients already taking corticosteroids during times of stress, the dosage may need to be increased.
  • There is an increased risk of infection when taking EMFLAZA. Tell the healthcare provider if the patient has had recent or ongoing infections or if they have recently received a vaccine. Medical advice should be sought immediately if the patient develops fever or other signs of infection. Patients and/or caregivers should be made aware that some infections can potentially be severe and fatal. Warn patients who are on corticosteroids to avoid exposure to chickenpox or measles and to alert their healthcare provider immediately if they are exposed.
  • EMFLAZA can cause an increase in blood pressure and water retention. If this occurs, dietary salt restriction and potassium supplementation may be needed.
  • There is an increased risk of developing a hole in the stomach or intestines in patients with certain stomach or intestine disorders when taking corticosteroids like EMFLAZA.
  • EMFLAZA can cause severe behavioral and mood changes. Seek medical attention from the health care provider if any behavioral or mood changes develop.
  • There is a risk of osteoporosis with prolonged use of EMFLAZA, which can lead to vertebral and long bone fractures.
  • EMFLAZA may cause cataracts or glaucoma and a health care provider should monitor for these conditions if corticosteroid therapy is continued for more than 6 weeks.
  • Immunizations should be up-to-date according to immunization guidelines prior to starting therapy with EMFLAZA. Live-attenuated or live vaccines should be administered at least 4 to 6 weeks prior to starting EMFLAZA. Live-attenuated or live vaccines should not be used in patients taking EMFLAZA.
  • EMFLAZA can cause serious skin rashes. Seek medical attention at the first sign of a rash.
  • Rare instances of anaphylaxis have occurred in patients receiving corticosteroid therapy, including EMFLAZA.

What should I tell my health care provider?

Tell the health care provider about all medical conditions, including if the patient:

  • is pregnant or planning to become pregnant. EMFLAZA® (deflazacort) can harm your unborn baby.
  • is breastfeeding or planning to breastfeed. EMFLAZA may appear in breastmilk and could affect a nursing child.

Certain medications can cause an interaction with EMFLAZA. Tell your healthcare provider of all the medicines you are taking, including over-the-counter medicines (such as insulin, aspirin or other NSAIDS), dietary supplements, and herbal products. Alternate treatment, dosage adjustment, and/or special test(s) may be needed during the treatment.

What are the side effects of EMFLAZA?

The most common side effects of EMFLAZA include facial puffiness or Cushingoid appearance, weight increased, increased appetite, upper respiratory tract infection, cough, frequent daytime urination, unwanted hair growth, central obesity, and colds. These are not all of the possible side effects of EMFLAZA. Call your doctor for medical advice about side effects.

To report an adverse event, please call 1-866-562-4620 or email at usmedinfo@ptcbio.com. You may also report side effects to FDA at 1-800-FDA-1088 or at www.fda.gov/medwatch.

Emflaza® is a registered trademark of PTC Therapeutics, Inc. Trademarks, registered or otherwise, are property of their respective owner(s). © 2021 PTC Therapeutics, Inc. All Rights Reserved.
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