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The ELEVIDYS impact

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ELEVIDYS slowed disease progression*

based on multiple measures of everyday movement

North Star Ambulatory Assessment

ELEVIDYS helped people

score higher on tests of everyday movements

Time to rise

ELEVIDYS helped people

get up from the floor faster

10-minute walk run

ELEVIDYS helped people

walk/run faster

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* Results were assessed at 1 year vs placebo and at 3 years vs external control. Additional assessments at 3 years are considered exploratory. Talk to your doctor to learn more.

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With ELEVIDYS, participants scored higher on tests that measure everyday movements

as measured by the average change in NSAA (North Star Ambulatory Assessment) score

1 year after ELEVIDYS
1 year after ELEVIDYS
3 years after ELEVIDYS
3 years after ELEVIDYS
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North Star Ambulatory Assessment

The NSAA looks at how well participants can perform 17 different everyday movements

Each movement is scored on a scale (0=the person cannot complete the movement at all; 1=they require help; 2=they can accomplish it independently). Scores are checked at different points to track changes over time. If a score increases by 1 or 2 points, it means a person maintained or improved their ability to perform that movement.

† Additional assessments are considered exploratory. Talk to your doctor to learn more.

All participants had similar characteristics (eg, age, physical ability) at the start of the study and were on stable doses of steroids.

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With ELEVIDYS, participants got up from the floor faster

as measured by the average change in time to rise from the floor

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1 year after ELEVIDYS
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Time to rise

Getting up from the floor faster after treatment suggests a delay in disease progression

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† Additional assessments are considered exploratory. Talk to your doctor to learn more.

All participants had similar characteristics (eg, age, physical ability) at the start of the study and were on stable doses of steroids.

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With ELEVIDYS, participants walked or ran faster

as measured by the average change in 10-meter walk/run time

1 year after ELEVIDYS
1 year after ELEVIDYS
3 years after ELEVIDYS
3 years after ELEVIDYS
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10-minute walk run

Walking/running faster after treatment suggests a delay in disease progression

† Additional assessments are considered exploratory. Talk to your doctor to learn more.

All participants had similar characteristics (eg, age, physical ability) at the start of the study and were on stable doses of steroids.

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Next up: Explore micro-dystrophin results

Now that you’ve seen how ELEVIDYS can slow disease progression, learn more about how it directly addresses the absence of dystrophin in Duchenne.

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Proven to produce ELEVIDYS micro-dystrophin

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3 months after ELEVIDYS
16 months after ELEVIDYS
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ELEVIDYS micro-dystrophin

The average increase in ELEVIDYS micro-dystrophin was consistent

People with Duchenne who are untreated typically have little to no dystrophin.

* Additional assessments are considered exploratory. Talk to your doctor to learn more.

† Results from Study 1 Part 1 (6 participants), Study 1 Part 2 (21 participants), Study 2 (40 participants), and Study 3 (17 participants). In Study 3, since people receiving placebo (14 participants) did not receive ELEVIDYS, they had 0% ELEVIDYS micro-dystrophin.

‡ Results from Study 3 (16 participants).

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Next up: Explore muscle loss data

Now that you’ve seen how ELEVIDYS directly addresses the absence of dystrophin in Duchenne, learn more about its impact on muscle loss.

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Colton received ELEVIDYS in 2024
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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 not recommended for individuals with:

  • Preexisting liver problems or liver infection because of the high risk of rapid serious liver injury and rapid liver failure
  • Recent vaccination (within 4 weeks of ELEVIDYS treatment)
  • Current or recent infections (within 4 weeks of ELEVIDYS treatment)
     

Important Safety Information

What is the most important information to know about ELEVIDYS?
Rapid Serious Liver Injury and Rapid Liver Failure

  • ELEVIDYS can increase certain liver lab test levels and cause rapid serious liver injury, rapid liver failure, and death. Patients with preexisting liver problems may be at higher risk.
  • Complication of blood clots in the blood vessel in the abdomen that helps carry blood from the intestines to the liver has happened.
  • Patients will receive oral corticosteroid medication before and after ELEVIDYS infusion and will need weekly blood tests to monitor liver function for 3 months or longer after treatment.
  • For at least 2 months following ELEVIDYS infusion, stay close to a healthcare facility that the doctor recommends.
  • Contact a doctor 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.

Serious Infection

  • Because patients will be taking corticosteroids as part of ELEVIDYS treatment, this may lower the ability of their immune system to fight infections and make it easier to get an infection. Getting an infection (like a cold, flu, stomach flu, ear infection, chest infection) before or after ELEVIDYS infusion could lead to more serious health problems, including death.
  • Contact a doctor right away if you notice any signs of infection, such as coughing, wheezing, sneezing, runny nose, sore throat, or fever.
  • Vaccinations should be completed at least 4 weeks before starting the corticosteroids that are part of the ELEVIDYS treatment.
  • ELEVIDYS should not be given to patients with an infection.

Inflammation of the Heart Muscle

  • Serious and life-threatening inflammation of the heart muscle has happened following ELEVIDYS infusion.
  • Patients will need weekly blood tests for a heart protein that can detect damage to the heart muscle cells for the first month after treatment.
  • Contact a doctor right away if the patient begins to experience chest pain and/or trouble breathing or shortness of breath.

Infusion-related Reactions

  • Infusion-related reactions, including hypersensitivity and serious allergic reactions (anaphylaxis), have happened during and after ELEVIDYS infusion.
  • Contact a doctor right away if you notice: 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.

Immune Response Affecting Muscles (Immune-mediated Myositis)

  • Immune response affecting muscles, including serious and life-threatening reactions, has happened in patients about 1 month after receiving ELEVIDYS infusion.
  • Contact a doctor immediately if the patient experiences any unexplained increased muscle pain, tenderness, or weakness, including trouble swallowing, breathing, or speaking.

Antibodies to ELEVIDYS

  • Patients need to have blood tests to ensure that they do not have antibodies that may prevent them from being able to receive ELEVIDYS. High levels of antibodies may keep the medicine from working as intended.
  • Treatment with ELEVIDYS is not recommended for patients who have high antibodies to the vector, the part of gene therapy used to deliver ELEVIDYS.

You should discuss the potential benefits and risks of ELEVIDYS with your doctor.

Who should not receive ELEVIDYS?
Individuals with a certain type of genetic mutation, called a deletion, involving any portion of or the entire exon 8 and/or exon 9 in the DMD gene, should not receive ELEVIDYS.

Are there any considerations for vaccination schedules and ELEVIDYS?
Patient vaccinations should be up to date with current immunization guidelines. Vaccinations should be completed at least 4 weeks before starting corticosteroids that are part of the ELEVIDYS treatment.

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 most common side effects of ELEVIDYS?
The most common side effects that occurred in patients treated with ELEVIDYS were vomiting, nausea, liver injury, fever, lower number of platelets (a kind of blood cell that helps you stop bleeding), and higher levels of heart protein that can detect damage to muscle cells in the heart. 

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, including Boxed Warning, and Medication Guide.