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Duchenne Muscular Dystrophy
Feb 25, 2020
sarepta.com
2:45
Hope for Duchenne Muscular Dystrophy
1K views
Dec 27, 2023
Facebook
Children's Hospital of The King's Daughters (C…
Duchenne Muscular Dystrophy (DMD)
Apr 28, 2018
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4-year-old boy with rare muscle condition sees 'new slate' with gene therapy
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New Duchenne Muscular Dystrophy treatment helps boy
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Tucson child receives first gene therapy treatment for Duchenne muscular dystrophy.
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New Future for Kids with Duchenne Muscular Dystrophy | Connor and Easton's Stories
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Jun 22, 2023
Facebook
Nationwide Children's Hospital
Duchenne muscular dystrophy (DMD) is an X-linked recessive genetic disease caused by mutations in the gene that encodes dystrophin, a large protein that plays an important role in the development of normal muscle fibers. The dystrophin gene is immense, spanning 2.5 million base pairs, and includes 79 exons and 78 introns. Many of the mutations that cause DMD produce premature stop codons, which bring protein synthesis to a halt, resulting in a greatly shortened and nonfunctional form of dystroph
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