ELEVIDYS voices

NARRATOR 

Important Safety Information 

What is ELEVIDYS? 

ELEVIDYS is a prescription gene therapy used to treat ambulatory individuals at least 4 years old with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene.   

ELEVIDYS is approved under accelerated approval for non-ambulatory patients at least 4 years old with DMD who have a confirmed mutation in the DMD gene. Accelerated approval allows for drugs to be approved based on a marker that is considered reasonably likely to predict a clinical benefit. ELEVIDYS treatment increased the marker, ELEVIDYS micro-dystrophin in skeletal muscle. Verification of a clinical benefit may be needed for ELEVIDYS to continue to be approved for non-ambulatory patients with DMD. 

Who should not receive ELEVIDYS? 

Individuals with certain types of mutations, any deletion in exon 8 and/or exon 9 in the DMD gene, should not receive ELEVIDYS.   

Please stay tuned for additional Important Safety Information at the end of this video and full Prescribing Information at ELEVIDYS.com.

Duchenne can vary in presentation, severity, and rate of progression. This is one person’s experience and may not be representative of all individuals with Duchenne. People who receive ELEVIDYS may have different experiences and varied treatment responses. Talk to your doctor about what may be right for you or your child.   

SAVANNAH 

My son Julius is six years old, and he lives with Duchenne muscular dystrophy.

At first it was me, my husband and Julius. For awhile there, we all got to enjoy our family of three, but it was always a goal to get my husband's four other kids. And then, during all that time, we so happened to got pregnant. Then made eight of us. Julius is very outspoken, very carefree and very funny. Naturally funny. I mean, he has me crying because he’s so funny.

MATTHEW

Julius is having the time of his life, no matter what. He’s always smiling. You know, he’s always just happy-go-lucky, funny guy. I know that my son is definitely the cornerstone of our family, a big part of our foundation, and definitely the glue that holds us together.

SAVANNAH

Muscular dystrophy is something that runs in my family. My twin brother, Derek, lived with Duchenne muscular dystrophy. I knew what the disease was like, and I wanted to take it head on as fast as I could. So, my neurologist let me know that there was a FDA-approved gene therapy that he qualified for. 

My initial reaction was excitement, also a little nerve-wracking. So that kind of pushed us in weighing the pros and the cons of getting this treatment for him. My mother and sister were excited because there was opportunities available now that weren't available 20 years ago.

MATTHEW

To be honest going into ELEVIDYS, it was a little scary, but seeing that the team of doctors involved in Julius’ care, specifically his neurologist, how involved they were. How much it’s meant to them, and it wasn’t just another patient, this is somebody who was becoming a part of their family, they’re becoming a part of our family.

In the days leading up to Julius’ treatment, there was a bit of anxiety that was growing, and every day that we got closer, it was a little more anxious. You go into the hospital room or the clinic and he’s there getting his IVs and he’s doing thumbs up and I’m here, like, terrified a little bit, you know. He’s the one telling me it’s okay. He’s the one making all this possible. He’s amazing. He really is.

SAVANNAH

Today Julius is doing well. We continue to notice changes. Recently, we had a big family outing and we went to a pizza arcade. He had his brothers and sister to actually run around and play along with so that was very nice.

MATTHEW

We’re looking forward to just Julius getting to be a six-year-old boy with his brothers and his sister and play and have fun and just enjoy life.

SAVANNAH 

I hope he can continue being the social butterfly that he is and continuing loving school, and loving his brothers and sisters, and just having a happy life.

MATTHEW

Our goal for Julius is for him to keep continuing to do the things he loves for as long as possible. 

SAVANNAH 

Since Julius was treated my feelings about Duchenne have just been more positive. We don’t know that the future holds with Duchenne, but we are happy with the path that we’re on.

MATTHEW

To think that Julius has an opportunity that his uncle didn't have is remarkable. In order to find comfort, we've come to the conclusion that people like Derek have helped lay a foundation for where we are today. Derek was a great step in allowing Julius to receive what he's received.

SAVANNAH 

It’s just nice to see that my son can have the opportunities that my twin brother, unfortunately, didn’t get to have, and, you know, to see his life just be a little bit different than what my brother had to deal with.

MATTHEW

Looking back at all the circumstances that led us to ELEVIDYS and his treatment, there’s no doubt that we would decide the same decision over again.

NARRATOR 

Please see full Prescribing Information at ELEVIDYS.com and continue watching for Important Safety Information. 

Important Safety Information

What is ELEVIDYS? 

ELEVIDYS is a prescription gene therapy used to treat ambulatory individuals at least 4 years old with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene.   

ELEVIDYS is approved under accelerated approval for non-ambulatory patients at least 4 years old with DMD who have a confirmed mutation in the DMD gene. Accelerated approval allows for drugs to be approved based on a marker that is considered reasonably likely to predict a clinical benefit. ELEVIDYS treatment increased the marker, ELEVIDYS micro-dystrophin in skeletal muscle. Verification of a clinical benefit may be needed for ELEVIDYS to continue to be approved for non-ambulatory patients with DMD. 

Who should not receive ELEVIDYS? 

Individuals with certain types of mutations, any deletion in exon 8 and/or exon 9 in the DMD gene, should not receive ELEVIDYS.   

What is the most important information to know about ELEVIDYS? 

Infusion-related reactions, including hypersensitivity and serious allergic reactions (anaphylaxis), have occurred during and after ELEVIDYS infusion. Symptoms may include fast heart rate, fast breathing, swollen lips, shortness of breath, nostrils widening, hives, red and blotchy skin, itchy or inflamed lips, rash, vomiting, nausea, chills, and fever. Your doctor will monitor you during and at least 3 hours after ELEVIDYS infusion. If an infusion-related reaction occurs, your doctor may slow or stop the ELEVIDYS infusion and provide additional medical treatment as needed. Contact a healthcare provider immediately if infusion-related symptoms occur.   

ELEVIDYS can increase certain liver enzyme levels and cause acute serious liver injury. Patients will receive oral corticosteroid medication before and after infusion with ELEVIDYS and will undergo weekly blood tests to monitor liver enzyme levels for 3 months after treatment. Contact a healthcare provider immediately if the patient’s skin and/or whites of the eyes appear yellowish or if the patient misses a dose of corticosteroid or vomits it up.   

Administration of ELEVIDYS may be delayed in patients who have acute liver disease until the condition is resolved or under control. Patients with preexisting liver impairment, chronic liver infection, or acute liver disease may be at higher risk of acute serious liver injury.   

Immune-mediated myositis (an immune response affecting muscles) was observed in patients with a deletion mutation in the DMD gene that is contraindicated.   

Patients with certain mutation deletions (in exons 1 to 17 and/or exons 59 to 71) may be at risk for a severe immune-mediated myositis reaction. 

Caregivers should contact a healthcare provider immediately if the patient experiences any unexplained increased muscle pain, tenderness, or weakness, including difficulty swallowing, breathing, or speaking, as these may be symptoms of myositis.   

Myocarditis (inflammation of the heart) has been observed within days following ELEVIDYS infusion. The patient’s doctor will conduct weekly blood tests for the first month after treatment to evaluate troponin-I (a cardiac protein that can detect damage to muscle cells in the heart). Caregivers should contact a healthcare provider immediately if the patient begins to experience chest pain and/or shortness of breath. More frequent monitoring may be required if the patient has cardiac symptoms. 

Patients need to have blood tests to ensure that they do not have antibodies that may prevent them from being able to receive ELEVIDYS, as introducing the gene therapy could increase the risk of a severe allergic reaction or prevent desired therapeutic levels. Treatment with ELEVIDYS is not recommended for patients who have high antibodies to the vector, the part of gene therapy used to deliver ELEVIDYS.   

Due to the need to follow a corticosteroid regimen, an infection (such as cold, flu, gastroenteritis [stomach flu], otitis media [ear infection], bronchiolitis [respiratory infection], etc.) before or after ELEVIDYS infusion could lead to more serious complications. Caregivers should contact a healthcare provider immediately if they see any symptoms suggestive of infection, such as coughing, wheezing, sneezing, runny nose, sore throat, or fever.   

Are there any considerations for vaccination schedules and ELEVIDYS?   

Patient vaccinations should be up to date with current immunization guidelines. Vaccinations should be received at least 4 weeks prior to starting the corticosteroid regimen that is required before receiving ELEVIDYS. 

Are there any precautions that need to be considered when handling a patient’s bodily waste? 

Vector shedding of ELEVIDYS occurs primarily through body waste. Patients and caregivers should use proper hand hygiene, such as hand washing when coming into direct contact with patient body waste. Place potentially contaminated materials that may have the patient’s bodily fluids/waste in a sealable bag and dispose into regular trash. Precautions should be followed for 1 month after ELEVIDYS infusion. 

What are the possible or likely side effects of ELEVIDYS? 

The most common side effects that occurred in patients treated with ELEVIDYS were vomiting, nausea, liver injury, fever, and decreased platelet counts.   

The safety information provided here is not comprehensive. Talk to the patient’s doctor about any side effects that bother the patient or that don’t go away. 

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782). 

Please see full Prescribing Information at ELEVIDYS.com. 

NARRATOR 

Important Safety Information 

What is ELEVIDYS? 

ELEVIDYS is a prescription gene therapy used to treat ambulatory individuals at least 4 years old with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene.   

ELEVIDYS is approved under accelerated approval for non-ambulatory patients at least 4 years old with DMD who have a confirmed mutation in the DMD gene. Accelerated approval allows for drugs to be approved based on a marker that is considered reasonably likely to predict a clinical benefit. ELEVIDYS treatment increased the marker, ELEVIDYS micro-dystrophin in skeletal muscle. Verification of a clinical benefit may be needed for ELEVIDYS to continue to be approved for non-ambulatory patients with DMD. 

Who should not receive ELEVIDYS? 

Individuals with certain types of mutations, any deletion in exon 8 and/or exon 9 in the DMD gene, should not receive ELEVIDYS.   

Please stay tuned for additional Important Safety Information at the end of this video and full Prescribing Information at ELEVIDYS.com.

Duchenne can vary in presentation, severity, and rate of progression. This is one person’s experience and may not be representative of all individuals with Duchenne. People who receive ELEVIDYS may have different experiences and varied treatment responses. Talk to your doctor about what may be right for you or your child.   

SYLVIA

My name is Sylvia. I’m married to Robert. We have three children, so we’ve got our hands full for sure. 

My son, Jason, is six and he lives with Duchenne. Jason is definitely a spitfire. He is the life of any party. And, you definitely know when he's in a room. He will make himself known for sure.

So, Jason absolutely loves dancing or singing, or even just, any type of activity that will get attention on him. I think one of the things Jason enjoys doing probably more than anything else is building Lego sets. I think that’s where he probably shines the most because, you know, he's able to be his creative self.

Once we got that Duchenne diagnosis, our family obviously had to process it. I knew immediately we needed to get the ball rolling and seeing a specialist that maybe knows a little bit more about Duchenne than possibly a doctor in our area, because we don’t have the specialty care down here.

So, we heard about ELEVIDYS through Jason's neurologist. Jason's doctor described very thoroughly what gene therapy, and specifically ELEVIDYS, would be doing. So, when they first told us about the possible side effects of ELEVIDYS, there was some nerves and some-- it wasn't doubt, but there was some moments where I was scared.

We did talk back and forth with the neurologist saying, “Hey, I have these concerns. Do you have concerns?” Once we had that lengthy conversation, we realized that this is definitely the next step for us. You know, Jason needs this gene therapy, and that’s the route we’re going to go.

Leading up to actual treatment day was kind of nerve-wracking because I was nervous. Like me, myself, I get nervous, but I would see Jason's reaction, and Jason's like, "When's my appointment? When's infusion day?" “He was excited because he goes, "I'm getting special medicine.”

He was so funny about everything and so excited about everything that it helped that process go, I think, a lot smoother then it would have been. The entire team there at the hospital were so helpful. The neurologist was so thorough in explaining the entire process of ELEVIDYS. That we were so comfortable because we knew there was going to be someone there with us every step of the way. It's all about support and we have an incredible support team when it comes to, not just his care, but anything in general.

Today, we're noticing a lot of positive changes with Jason. He's been looking forward to having his birthday party in his favorite pizza place where he can play all his arcade games with his friends and cousins.

ELEVIDYS is not a cure. I know Jason still has Duchenne. I don’t know what the future holds with Duchenne, but I’m happy with the path we’re on. Would we choose ELEVIDYS again if we were given the chance? You know, this is something that, as a family, we decided was definitely the right choice for us.

In the region that we live, some people may call it southmost Texas. You can definitely say that it’s, you know, the Latino culture, the Latino community is definitely prominent in our area. Spanish is spoken much more than English. There might be a language barrier in educating the community about Duchenne. 

My advice is you need to advocate for yourself and you need to advocate for your child. If that means pushing your medical team, and asking for answers, then do that. Maybe it truly is the Latina in me where I get spicy and I just fight for what I think is right.

So, looking ahead, like looking into the future my dreams for Jason are just for him to live life to the fullest, and he honestly does that.

NARRATOR 

Please see full Prescribing Information at ELEVIDYS.com and continue watching for Important Safety Information. 

Important Safety Information

What is ELEVIDYS? 

ELEVIDYS is a prescription gene therapy used to treat ambulatory individuals at least 4 years old with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene.   

ELEVIDYS is approved under accelerated approval for non-ambulatory patients at least 4 years old with DMD who have a confirmed mutation in the DMD gene. Accelerated approval allows for drugs to be approved based on a marker that is considered reasonably likely to predict a clinical benefit. ELEVIDYS treatment increased the marker, ELEVIDYS micro-dystrophin in skeletal muscle. Verification of a clinical benefit may be needed for ELEVIDYS to continue to be approved for non-ambulatory patients with DMD. 

Who should not receive ELEVIDYS? 

Individuals with certain types of mutations, any deletion in exon 8 and/or exon 9 in the DMD gene, should not receive ELEVIDYS.   

What is the most important information to know about ELEVIDYS? 

Infusion-related reactions, including hypersensitivity and serious allergic reactions (anaphylaxis), have occurred during and after ELEVIDYS infusion. Symptoms may include fast heart rate, fast breathing, swollen lips, shortness of breath, nostrils widening, hives, red and blotchy skin, itchy or inflamed lips, rash, vomiting, nausea, chills, and fever. Your doctor will monitor you during and at least 3 hours after ELEVIDYS infusion. If an infusion-related reaction occurs, your doctor may slow or stop the ELEVIDYS infusion and provide additional medical treatment as needed. Contact a healthcare provider immediately if infusion-related symptoms occur.   

ELEVIDYS can increase certain liver enzyme levels and cause acute serious liver injury. Patients will receive oral corticosteroid medication before and after infusion with ELEVIDYS and will undergo weekly blood tests to monitor liver enzyme levels for 3 months after treatment. Contact a healthcare provider immediately if the patient’s skin and/or whites of the eyes appear yellowish or if the patient misses a dose of corticosteroid or vomits it up.   

Administration of ELEVIDYS may be delayed in patients who have acute liver disease until the condition is resolved or under control. Patients with preexisting liver impairment, chronic liver infection, or acute liver disease may be at higher risk of acute serious liver injury.   

Immune-mediated myositis (an immune response affecting muscles) was observed in patients with a deletion mutation in the DMD gene that is contraindicated.   

Patients with certain mutation deletions (in exons 1 to 17 and/or exons 59 to 71) may be at risk for a severe immune-mediated myositis reaction. 

Caregivers should contact a healthcare provider immediately if the patient experiences any unexplained increased muscle pain, tenderness, or weakness, including difficulty swallowing, breathing, or speaking, as these may be symptoms of myositis.   

Myocarditis (inflammation of the heart) has been observed within days following ELEVIDYS infusion. The patient’s doctor will conduct weekly blood tests for the first month after treatment to evaluate troponin-I (a cardiac protein that can detect damage to muscle cells in the heart). Caregivers should contact a healthcare provider immediately if the patient begins to experience chest pain and/or shortness of breath. More frequent monitoring may be required if the patient has cardiac symptoms. 

Patients need to have blood tests to ensure that they do not have antibodies that may prevent them from being able to receive ELEVIDYS, as introducing the gene therapy could increase the risk of a severe allergic reaction or prevent desired therapeutic levels. Treatment with ELEVIDYS is not recommended for patients who have high antibodies to the vector, the part of gene therapy used to deliver ELEVIDYS.   

Due to the need to follow a corticosteroid regimen, an infection (such as cold, flu, gastroenteritis [stomach flu], otitis media [ear infection], bronchiolitis [respiratory infection], etc.) before or after ELEVIDYS infusion could lead to more serious complications. Caregivers should contact a healthcare provider immediately if they see any symptoms suggestive of infection, such as coughing, wheezing, sneezing, runny nose, sore throat, or fever.   

Are there any considerations for vaccination schedules and ELEVIDYS?   

Patient vaccinations should be up to date with current immunization guidelines. Vaccinations should be received at least 4 weeks prior to starting the corticosteroid regimen that is required before receiving ELEVIDYS. 

Are there any precautions that need to be considered when handling a patient’s bodily waste? 

Vector shedding of ELEVIDYS occurs primarily through body waste. Patients and caregivers should use proper hand hygiene, such as hand washing when coming into direct contact with patient body waste. Place potentially contaminated materials that may have the patient’s bodily fluids/waste in a sealable bag and dispose into regular trash. Precautions should be followed for 1 month after ELEVIDYS infusion. 

What are the possible or likely side effects of ELEVIDYS? 

The most common side effects that occurred in patients treated with ELEVIDYS were vomiting, nausea, liver injury, fever, and decreased platelet counts.   

The safety information provided here is not comprehensive. Talk to the patient’s doctor about any side effects that bother the patient or that don’t go away. 

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782). 

Please see full Prescribing Information at ELEVIDYS.com.