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What types of DMD can Exondys 51 be used for?

Medically reviewed by Carmen Pope, BPharm. Last updated on Feb 23, 2023.

Official answer

by Drugs.com

Exondys 51 can only be used by patients with DMD who have a confirmed mutation in their dystrophin gene that can be treated by skipping exon 51. This works out to be about 13% of people with DMD.

Ask your doctor about genetic testing and if you haven’t already done so, ask for a genetic test for your child. This test can identify the genetic mutation and if it is amenable to treatment with Exondys 51. Genetic testing methods have changed with time, so ask your doctor to order a new test if the test was done years ago.

References
  • Identifying your child’s mutation. Sarepta https://www.exondys51.com/who-is-it-for/know-your-duchenne-genetic-mutation

Read next

The new drugs approved by the FDA for the treatment of Duchenne Muscular Dystrophy (DMD) are Amondys 45, Viltepso, Vyondys 53, Exondys 51, Elevidys, Emflaza and Duvyzat. They include classes such as antisense oligonucleotides, a glucocorticoid, a gene therapy and a histone deacetylase (HDAC) inhibitor. Continue reading

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

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. Continue reading

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