Family Takes Advantage of Temporary Approval of Exondys 51


A family from Torrington, Connecticut is taking advantage of the FDA’s temporary approval of the muscular dystrophy drug Exondys 51 (or Eteplirson). In a report from, mom Mel Kelly had joined an MD movement that applied pressure on the FDA to approve the drug.

Find out more about the approval of Exondys 51 here.

When the FDA gave the drug a temporary accelerated approval, Kelly immediately started her two sons on the treatment. Twenty-year-old Jacob and his 16-year-old brother Liam both suffer from Duchenne muscular dystrophy. They began the weekly IV-administered treatment in December and after just two months the family is starting to see an improvement.

Liam is now able to hug his mom around the neck and clasp his hands together, something he hadn’t been able to do for a long time.

Kelly hopes that the full FDA approval of the drug happens quickly so it becomes accessible to more muscular dystrophy families and can start to change more lives.

Find out about other treatment options for Duchenne muscular dystrophy.

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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