Exondys 51 Treatment for Duchenne Muscular Dystrophy


Exondys 51 is a new treatment for Duchenne muscular dystrophy that was granted fast-tracked approval in the U.S. by the FDA in September 2016.

Ohio boy to start Exondys 51 treatment for Duchenne muscular dystrophy.

Injections of the drug could lessen the impact of muscle weakness and slow progression of the disease in certain muscular dystrophy patients who have a mutation in the dystrophin gene, which accounts for around 13 percent of Duchenne patients.

Exondys 51 is an exon-skipping drug which allows it to restore production of the dystrophin protein, which is essential for muscle building.

The FDA has allowed the fast-tracked approval of Exondys 51 so that further clinical trials may be extended and more evidence gathered about the benefits of the drug. However, if no significant evidence of clinical benefit is found, the FDA could withdraw its approval. Find out more about Exondys 51 here. 

CareDuchenne seeks treatment for the 87 percent of patients not helped by Exondys 51

Muscular Dystrophy News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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  1. PADMINI says:

    My son is affected by DMD. his age is 8. is any possible for his treatment pls tell us.This Exondys 51 treatment is possible for him. Tell about this treatment briefly & send the details to my
    e-mail id.

  2. javad says:

    Hi there.im looking for cure for my son.
    he’s affected with DMD and has hemizygote deletion in exon 52 of dystrophin gene.
    Please help me out .
    Is there any cure for this type of DMD?
    please contant me to send his tests.thanks

  3. Mola Ageze says:

    Please I’m desperately seeking help for my 9 years old son.He is not diagnosed with DMD, but his signs are leading to that direction. He will have a Muscle Biopsy test on Jan 2018. Please help if there is an early treatment for him before it gets worst.

  4. Mohsen says:

    Hi. My cusin is affected by dmd with deletion in exons 45-50.please give me information about its treatment via my email.

  5. eugueni gomez galan says:

    hello my son lives in Brazil and the treatment was suspended by the goverment. I would like to know how do i find information about that. I live in Green Bay Wisconsin and I need help about that please!

  6. Rutu says:

    Hi my son 9yrs old diagnosed with DMD ,he is taking steroids low dose but he is getting weaker and delay growth period.please advise me for any appropriate treatment.Please help me for any treatment or any thoughts.I am so worried everyday.

  7. Vishal says:

    My son is suffering with DMD. Age-13 years. Exon 45. Pl confirm treatment or any updation regarding skipping of exon

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