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What You Need to Know About Duchenne Muscular Dystrophy Gene Therapy
Gene therapy is poised to revolutionize how Duchenne is treated, but there are still some problems with this approach that need to be solved.
What Causes Duchenne Muscular Dystrophy?
Different kinds of mutations on the same gene can all lead to a lack of dystrophin, a protein that’s critical for long-term muscle function.