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New step toward treatment for Duchenne muscular dystrophy

This press release is also available in French.

Quebec City, June 8, 2006--The team led by Dr. Jacques P. Tremblay, a researcher with the Human Genetics Department at Quebec City's Centre Hospitalier Universitaire de Qubec (CHUQ) and professor with Universit Laval's Faculty of Medicine, has taken an important step toward a cure for Duchenne muscular dystrophy. After performing the first successful muscle cell transplant in young patients two years ago, professor Tremblay announced today that eight out of the nine patients have shown promising results.

Duchenne muscular dystrophy is a progressive weakening and degeneration of the muscles, caused by a mutation in a gene coding for a protein called dystrophin. This protein is absent in the muscles of people affected with the disorder. With the healthy muscle cells grafted by the team of researchers, a considerable proportion of patients' muscle fibers are now able to synthesize the missing protein. Analyses showed that between 4% and 26% of muscle cells in the cubic centimeter of treated muscle eventually produce the protein produced by the fusion of the donor cells. The results are published in the latest issue of the Journal of Neuropathology and Experimental Neurology. Dr. Tremblay's team is the first in the world to achieve this level of dystrophin expression in so many patients, and in such a high percentage of fibers.

Duchenne muscular dystrophy is a hereditary disease affecting one boy in 3,500. The first signs appear in childhood, between the ages of two and five. Its victims are confined to a wheelchair by their early teens. The disease progressively weakens all muscles, notably respiratory muscles, compromising life expectancy. Most sufferers die between the ages of 20 and 30. Today, there is no treatment--only rehabilitation, surgical procedures and prednisone to enhance patients' quality of life.
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Contact: Martin Guay
martin.guay@dap.ulaval.ca
418-656-3952
Universit Laval
8-Jun-2006


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