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



This video is based on the personal experience of families with boys taking emflaza® (deflazacort). Please speak to a healthcare provider before making treatment decisions.


DMD=Duchenne Muscular Dystrophy

Sarah: I'm Sarah.

Dan: I'm Dan.

Sarah: And we have four boys. AJ, Ben, Caleb, and Duncan. AJ is 21, Ben is 19, Caleb is eight, and Dunky is six. They like to go swimming, we love to travel with the boys, we love to go out to eat.


Sarah: Once we got home and, were able to kind of digest what we had been diagnosed with, we instantly started to advocate and just live differently, to be honest. So, started to kind of create some of the memories because we were worried that, we didn't know how long we would have them for.

What Treatment Approach Did Your Sons' Care Team Suggest?

Dan: Talking to doctors and the community members, we felt like we had figured out our choice and went down the right road.

Sarah: So we started Caleb first on EMFLAZA and then, subsequently, Dunky just a few weeks after.

What Do You Do To Stay Healthy?

Sarah: We love to make smoothies, we're a big smoothie family, I would say almost every day.

How Have Other Parents Going Through a Similar Journey Helped?

Sarah: I would say my friendship circle has changed. Not like we're just friends with other Duchenne families, but we get each other. And that's really helpful.

A Closing Thought on the Here & Now

Sarah: Living in the moment because we don't know how much time we have. I would say we feel lucky to be able to kind of live differently, with a purpose, with a mission.

Dan: And we continue to dig for different things that help them manifest that giggling and laughing and being silly and we don't ever know where we're going to find it next.




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.

PTC Cares™ is a trademark of PTC Therapeutics, Inc. Emflaza® is a registered trademark of PTC Therapeutics, Inc. © 2022 PTC Therapeutics, Inc. All Rights Reserved.
US-EMF-0376 06/22