Important Safety Information
Prescribing Information

Transcript

Making a Switch: Chistopher's Treatment Journey

Sima: Hello, my name is Sima Perez and I am the mother of Christopher Perez. Christopher is 16 years-old, and he has Duchenne muscular dystrophy. We live in Long Beach, California. My daughter is Rebecca Perez, she is 18 years-old, and my husband Bernie. And we have two cats, Tobey and Tomás.

Sima: Christopher is a very smart guy. He is going to be a senior this year. He's also working—he has a job, his very first job at the local community center and he's really excited about that.

Sima: I can recall early conversations I had with our neurologist was when Christopher was still walking. Christopher was 6 to 7 years-old. The conversation came up, in the sense that we wanted to prolong his gait, we wanted to prolong him being ambulatory. And so the discussion was around drugs and steroids, to be more specific.

Sima: And so we talked about the side effects, we discussed the benefits and we both thought at the time that it would be a great idea for Christopher to begin the steroid medicine, because the benefits outweigh the risks.

Sima: At about 9 and a half years-old, Christopher stopped walking. And we originally thought that it would not be beneficial anymore to have him be on steroids. And so the neurologist and I had a conversation and she said, “on the contrary, we feel that Christopher needs to be on corticosteroids forever.” And I didn't understand though why. The neurologist told us that we needed to preserve Christopher's muscle and support the lung function for him for as much as possible.

Sima: Christopher was on prednisone for a couple of years. We noticed some changes, and then we decided to transition and move him on to EMFLAZA.

Sima: The process felt really easy and it continues to be easy. I do get, quite often, phone call reminders that it's time to schedule his order and/or delivery.

Sima: What stands out the most to me is that our Case Manager, Rick, is very friendly. And he is very willing to answer any questions and he always tells me “if I don't know the answer, I'll find someone who can get you the answer,” but for the most part it's a very easy process.

Rick: The first time me and Sima connected was a couple years back. Yes, she was going through—Chris was going through some issues with coverage and obtaining his refill for that month. My first impression of Sima was wow, she knows her stuff. She was aware of insurances. She kind of knew general next steps so it made it easier to solve the problem.

Rick: Chris was running out of medication, so we were working through obviously figuring out what was going on and also, “hey, Chris needs medicine.” And, “how can we figure this out within a week's time?”

Rick: Some of the services that I've been able to provide to Sima and a lot of our families is the basics; obviously verifying insurance, reviewing out-of-pocket costs, working very closely with the specialty pharmacies and the neurologist and providers and their staff. And then we also help connect our families with other families through our Peer Navigator Program. So yeah, there's a lot on the surface and then once patients are on therapy, you know, we're checking in and obviously continuing that relationship with our families.

Rick: I can speak for the team when I say that the group here at PTC Cares, we do care about our families.

Sima: Christopher wants to go to college; Christopher is excited about the future. As a mother with a son that has muscular dystrophy, it makes me very happy to know that he is looking forward to bigger and better things for him, and I am, you know, his number one supporter and I will be with him every step of the way.

To get connected with a Peer Navigator, please contact us at peernavigator@ptcbio.com or (866) 282-5878.

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.

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.
US-EMF-0391 09/22