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Cure Insight for DMD

According to the researcher Dr. Jacques P. Tremblay who is in the Human Genetics Department at Quebec City's Centre Hospitalier Universitaire de Québec (CHUQ) // and professor with Université Laval's Faculty of Medicine muscle cell transplant is one of the important alternatives to cure Duchenne muscular dystrophy. He and his team of researchers conducted the muscle cell transplant in young patients in 2004. The results were that eight out of the nine patients showed improvement. 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. New clinical trial: seeking ten patients Based on these results, professor Tremblay has received authorization to conduct a second round of clinical trials.
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