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12:00 AM
Jan 07, 2008
 |  BioCentury  |  Product Development

Addition by subtraction

Existing treatments for Duchenne muscular dystrophy are only palliative, and survival beyond the early 30s is rare. Prosensa B.V. believes its exon skipping technology can target the cause of the disease and halt its progression by modulating pre-mRNA processing so that a functional form of dystrophin is produced. Last month, the company published data from a pilot trial in The New England Journal of Medicine, showing PRO051 restored dystrophin expression in the muscle fibers of four patients.

Duchenne muscular dystrophy (DMD) is a genetic neuromuscular degenerative disease caused by errors or deletions in the dystrophin gene that result in erroneous coding for the protein. The resulting mRNA is "out of frame," meaning it cannot be read by the cell's machinery and dystrophin cannot be produced....

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