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Exon Skipping Drug Prevents Muscle Wasting and Maintains Muscle Function in Severely Affected, Dystrophin Deficient Mice
AVI BioPharma, 10/21/09
An exon skipping PPMO has demonstrated dramatic effects in the prevention and treatment of severely affected, dystrophin and utrophin–deficient mice, preventing severe deterioration of the treated animals and extending their lifespan. These findings were published online today in the journal Molecular Therapy and support the promise of this therapeutic approach for the treatment of Duchenne muscular dystrophy.
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