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.
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.
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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