DUCHENNE IS A RARE DISEASE THAT WEAKENS MUSCLES

Duchenne muscular dystrophy (DMD) is a disorder in which patients are born with a gene mutation or defect that keeps their bodies from creating dystrophin, a protein that protects muscles from damage.¹

Dystrophin works to protect muscles from injury

Dystrophin is a protein found in muscles and works with other proteins to make sure muscles are protected from injury as they expand and contract. Because patients affected by Duchenne have a mutation that affects the genetic structure of dystrophin, it isn't able to do its job properly. As a result, patients experience muscle weakening in their legs and arms. ^2-4

BOYS ARE MORE LIKELY TO BE AFFECTED BY DUCHENNE

The dystrophin gene is carried by the X-chromosome. Girls have two X-chromosomes, so if one of the chromosomes is affected by the dystrophin mutation that causes DMD, they may not develop DMD symptoms. Girls who have one X-chromosome affected by a dystrophin mutation but do not have DMD symptoms are known as DMD carriers. ^1,5

On the other hand, boys only have one X-chromosome which means if it is affected by a dystrophin mutation they do not have another X-chromosome to protect them from developing symptoms of DMD. This is why boys are more likely than girls to be affected by Duchenne. ^1,5

It affects about 1 in 3,600 - 6,000 live male births. ^6-9

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REFERENCES

1. Bushby K, Finkel R, Birnkrant DJ, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol. 2010;9(1):77-93. 2. Signs and symptoms. Parent Project Muscular Dystrophy website. http://www.parentprojectmd.org/site/PageServer?pagename=Understand_about_signs. Accessed May 3, 2018. 3. DMD gene. U.S. National Library of Medicine website. https://ghr.nlm.nih.gov/gene/DMD. Accessed April 9, 2018. 4. Duchenne and Becker muscular dystrophy. U.S. National Library of Medicine website. https://ghr.nlm.nih.gov/condition/duchenne-and-becker-muscular-dystrophy#genes. Accessed April 9, 2018. 5. Genetic causes. Parent Project Muscular Dystrophy website. https://www.parentprojectmd.org/about-duchenne/what-is-duchenne/genetic-causes. Accessed May 8, 2018. 6. Van Ruiten H, Straub V, Bushby K, et al. Improving recognition of Duchenne muscular dystrophy: a retrospective case note review. Arch Dis Child. 2014;99:1074-1077. 7. Goemans N, Kirschner J, Mercuri E. New perspectives in the management of Duchenne muscular dystrophy. European Neurological Review. 2014;9(1):78-82. 8. Mendell JR, Lloyd-Puryear M. Report of MDA muscle disease symposium on newborn screening for Duchenne muscular dystrophy. Muscle Nerve. 2013;48(1):21-26. 9. Birnkrant DJ, Bushby K, Bann CM, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. Lancet Neurol. 2018;17(3):251-267.

INDICATION & IMPORTANT SAFETY INFORMATION FOR EMFLAZA® (deflazacort)

INDICATION

EMFLAZA^® is indicated for the treatment of Duchenne muscular dystrophy in patients 5 years of age and older.

IMPORTANT SAFETY INFORMATION

Contraindication: Do not use if you are allergic to deflazacort or any of the inactive ingredients in EMFLAZA.

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.

•	Hyperglycemia: Corticosteroids can increase blood glucose, worsen pre-existing diabetes, predispose those on long-term treatment to diabetes mellitus, and may reduce the effect of anti-diabetic drugs. Monitor blood glucose at regular intervals. For patients with hyperglycemia, anti-diabetic treatment should be initiated or adjusted accordingly.
•	Increased Risk of Infection: Tell your healthcare provider if you have had recent or ongoing infections or if you have recently received a vaccine or are scheduled for a vaccination. Seek medical advice at once should you develop fever or other signs of infection, as some infections can potentially be severe and fatal. Avoid exposure to chicken pox or measles, but if you are exposed, medical advice should be sought without delay.
•	Alterations in Cardiovascular/Kidney Function: EMFLAZA can cause an increase in blood pressure, salt and water retention, or a decrease in your potassium and calcium levels. If this occurs, dietary salt restriction and potassium supplementation may be needed.
•	Behavioral and Mood Disturbances: There is a potential for severe behavioral and mood changes with EMFLAZA and you should seek medical attention if psychiatric symptoms develop.
•	Effects on Bones: There is a risk of osteoporosis or decrease in bone mineral density with prolonged use of EMFLAZA, which can potentially lead to vertebral and long bone fractures.
•	Effects on Growth and Development: Long-term use of corticosteroids, including EMFLAZA may slow growth and development in children.
•	Ophthalmic Effects: EMFLAZA may cause cataracts or glaucoma and you should be monitored if corticosteroid therapy is continued for more than 6 weeks.
•	Vaccination: The administration of live or live attenuated vaccines is not recommended. Killed or inactivated vaccines may be administered, but the responses cannot be predicted.
•	Serious Skin Rashes: Seek medical attention at the first sign of a rash.
•	Drug Interactions: 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.

Common side effects that could occur with 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.

Please see the accompanying full Prescribing Information

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