Researchers are looking for a cure for Duchenne muscular dystrophy

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COLUMBIA - Thursday, a new technology was introduced at Missouri's Tech Expo could help find a cure for Duchenne muscular dystrophy, DMD, in the near future. 

DMD is a genetic disorder that causes progressive muscular weakness and irreversible deterioration of muscles. As of right now, there is no cure. 

The CEO and Founder of Solid Biosciences, Ilan Ganot, introduced a gene delivery technology that is looking to change that.

Ganot said the gene therapy approach is an attempt to effectively replace a defective gene with a new copy of a functional gene. 

He said his company has partnered with a group of individuals at the University of Missouri, led by Dongsheng Duan, a muscle biologist.

Ganot said Duan has lots of experience with animals related to this disease.

"This technology could well translate into a real change for this disease for basically every patient," Ganot said. 

Ganot said DMD affects one in 3-4 thousands newborn boys. He also said there usually is no family history of the disease.  

He said people who have DMD are usually wheelchair bound by their teenage years and typically die in their 20s.  

Ganot's son, Eytani, was diagnosed with DMD three years ago on this day. He said the diagnosis was very unexpected. He said at the time his son was a little slow, but not very different from his age group. He said overtime, the gap between his son and his son's age group has significantly widened.  

Ganot gave up his career as an investment banker to start his company in hopes to find a treatment, or even a cure. 

Ganot's named his business, Solid Biosciences, after his son. Eytani, means "solid" in Hebrew. 

Girls are typically not affected by the disease. Ganot said the disease is an X chromosome defect. Since girls have two X chromosomes, they can carry the gene but not become sick. On the other hand, if the X chromosome in a boy is affected, they will become sick. 

  

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