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Please see the Important Safety Information and full Prescribing Information here.

REACH OUT TO THE DUCHENNE COMMUNITY

The Duchenne community can offer support to you and your family in many different ways.

ADVOCACY GROUPS ARE READY TO HELP

We know that there is more to treatment than just taking care of symptoms. You and your family also have your own individual interests and support needs. Advocacy groups play an important role in helping you connect and bond with other people affected by Duchenne as well as giving patients a public voice.


Advocacy groups in your community:

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Cure Duchenne
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Muscular Dystrophy Association (MDA)
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Parent Project Muscular Dystrophy (PPMD)

ppmd Certified Duchenne Care centers and mda care centers

PPMD Certified Duchenne Care Centers and MDA Care Centers provide comprehensive care and services specifically for people living with Duchenne muscular dystrophy. The goal of these centers is to make comprehensive care available and accessible to as many patients with Duchenne and their families as possible. The PPMD Certified Duchenne Care Centers and MDA Care Centers can be found on their website: parentprojectmd.org/carecenters and mda.org/disease/duchenne-muscular-dystrophy.

PTC does not warrant, whether expressed or implied, that the list of centers is accurate or complete. PTC does not recommend or endorse any center, hospital, or healthcare professional included above for any purpose, nor is the list to verify a center’s, hospital’s, or specific healthcare professional’s credentials or qualifications. It is your responsibility to select a center, hospital, and/or healthcare professional appropriate for your needs and you assume full responsibility for your interactions with any center, hospital, or healthcare professional you contact through the center list. PTC is not responsible for the medical care or advice given by any center, hospital, or healthcare professional you choose to contact.

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.

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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.
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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.
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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.
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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.
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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.
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Effects on Growth and Development: Long-term use of corticosteroids, including EMFLAZA may slow growth and development in children.
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Ophthalmic Effects: EMFLAZA may cause cataracts or glaucoma and you should be monitored if corticosteroid therapy is continued for more than 6 weeks.
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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.
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Serious Skin Rashes: Seek medical attention at the first sign of a rash.
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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

For medical information, product complaints, or to report an adverse event, please call 1‑866‑562‑4620 or email at usmedinfo@ptcbio.com.

You may report adverse events to FDA at 1‑800‑FDA‑1088 or www.fda.gov/medwatch.

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PTC Cares™ and Navigating Duchenne™ are trademarks of PTC Therapeutics, Inc.
Emflaza® is a registered trademark of PTC Therapeutics, Inc.
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