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

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