Important Safety Information
Prescribing Information

Video library

Understanding and managing DMD can be difficult on your own. The videos below feature stories from families going through similar journeys along with other useful information. Watch them all to see if anything they have to say could help you and your family.

Here & now with the LoSapios

Mike has two sons with DMD. Here's how he makes sure they're making the most of the here & now.

Here & now with the Kasners

Sarah and Dan work together to care for their two sons with DMD. Watch how they try to have fun and enjoy life as much as they can.

Making A Switch: Christopher's Treatment Journey

Sima cares for her son every day with the help of her family. Hear how she works with the DMD community and PTC Cares™ along the way.

Haciendo un cambio: la historia de tratamiento de Christopher

Sima cuida de su hijo cada día con la ayuda de su familia. Escuche cómo trabaja con la comunidad de DMD y PTC Cares™ a lo largo del proceso.

A real-world story

Hear from a family as they discuss their experience with making treatment decisions.

Challenging Expectations

Find out how one DMD mom, and Peer Navigator, helps her son pursue a normal life by adapting activities to his abilities.

Making the Grade

Hear from a mother caring for her son with DMD on how she's setting him up for success in school.

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Indication & Important Safety Information for EMFLAZA® (deflazacort)

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 Acute adrenal insufficiency can occur if corticosteroids are withdrawn abruptly, and can be fatal.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.

Indication & Important Safety Information for EMFLAZA® (deflazacort)

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.

Prescription Start Form

Indication & Important Safety Information for EMFLAZA® (deflazacort)

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.