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The resulting dystrophin isn't perfect, but good enough to help stabilize muscles, researchers say.
WSJ: New Muscular Dystrophy Drugs Offer Hope
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By skipping a single missing letter, the drug makes a shorter but effective version of dystrophin.
FORBES: In A First, An Experimental Drug May Help Boys With Muscular Dystrophy
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The researchers bred mice that produced no dystrophin, or another related protein called utrophin.
BBC: Lab work
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The new treatment is based on the theory that excess integrin compensates for the lack of dystrophin.
BBC: Lab work
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For sick kids that might mean producing just enough of the missing dystrophin to stop their muscles from withering.
FORBES: Stopping the Nonsense
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Duchenne patients have more of the integrin, but fail to produce another protein, dystrophin, which is also required for healthy muscles.
BBC: Lab work
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Duchenne is caused by misspellings in the gene that tells the body to produce a protein called dystrophin, a glue that helps hold muscle tissue together.
FORBES: Stopping the Nonsense
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Boys with Duchenne muscular dystrophy lack this protein, called dystrophin.
WSJ: New Muscular Dystrophy Drugs Offer Hope
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It works because many cases of muscular dystrophy are caused by mutations in the dystrophin gene (incidentally, one of the longest genes in the body) that make the entire genetic code for the protein shift by one letter.
FORBES: In A First, An Experimental Drug May Help Boys With Muscular Dystrophy
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The number of muscle fibers that tested positive for dystrophin increased 47% in patients who had been receiving eteplirsen since the beginning of the study and 38% in the patients who started on placebo and switched to eteplirsen after 24 weeks.
FORBES: In A First, An Experimental Drug May Help Boys With Muscular Dystrophy