Is there a specific age range for which Exondys 51 will work?
There is currently no specific age range specified for using Exondys 51, but a two-part Phase 3 study scheduled to run until 2026 has recruited boys and young men aged 4 though 21 who meet the study criteria. Previous studies have only investigated boys up to age 19 years. Symptoms of Duchenne muscular dystrophy (DMD) usually appear between 3 and 5 years of age and the condition primarily affects boys at a rate of about 1 in every 3,600 male infants born. If you have an older child, the best thing you can do is talk to your doctor about Exondys 51 treatment.
References
- Madesen A. Top 5 FAQs: Eteplirsen (Exondys 51) for DMD Treatment Sept 2016. MDA News. https://strongly.mda.org/eteplirsen-exondys-51-dmd-treatment-top-5-frequently-asked-questions/
- Exondys 51. Sarepta https://www.exondys51.com/faqs
Read next
What are the new drugs for DMD (Duchenne muscular dystrophy)?
The new drugs approved by the FDA for the treatment of Duchenne Muscular Dystrophy (DMD) are Agamree, Amondys 45, Duvyzat, Elevidys, Emflaza, Exondys 51, Viltepso, and Vyondys 53. They include classes such as antisense oligonucleotides, glucocorticoid (corticosteroids), a gene therapy and a histone deacetylase (HDAC) inhibitor. Continue reading
Is Exondys 51 a type of gene therapy?
Exondys 51 is not technically gene therapy, but it does treat people with Duchenne muscular dystrophy who have a confirmed genetic mutation in their dystrophin gene which means they are unable to make the protein dystrophin. Dystrophin is needed for muscle repair. Without it, muscle cells become damaged and weakened over time, which can affect voluntary movement, such as walking. Continue reading
How is Exondys 51 administered?
Exondys 51 is administered once a week as a 35 to 60-minute intravenous infusion by your healthcare provider. The correct dosage of Exondys 51 is drawn up and then diluted in an infusion bag containing 0.9% sodium chloride to make a total volume of 100-150 mL. The diluted solution is then administered into one of your veins via an in-line 0.2-micron filter. Continue reading
Related medical questions
- What types of DMD can Exondys 51 be used for?
- How much does Elevidys cost?
- How does Vyondys 53 work?
- How long does it take for Viltepso to work?
- How long does it take for Vyondys 53 to work?
- How is Vyondys 53 administered?
- What type of drug is Emflaza, how does it work?
Drug information
Related support groups
- Exondys 51 (5 questions, 3 members)
- Eteplirsen (4 questions, 3 members)
- Duchenne Muscular Dystrophy (11 questions, 4 members)