Researchers are looking for a cure for Duchenne muscular dystrophy

4 years 7 months 1 week ago Thursday, October 15 2015 Oct 15, 2015 Thursday, October 15, 2015 12:42:00 PM CDT October 15, 2015 in News
By: Haley Hughes, KOMU 8 Reporter
loading

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. 

  

More News

Grid
List
JEFFERSON CITY – During his daily COVID-19 briefing Thursday, Governor Mike Parson announced an extension of the phase one reopening... More >>
28 minutes ago Thursday, May 28 2020 May 28, 2020 Thursday, May 28, 2020 8:42:00 PM CDT May 28, 2020 in News
COLUMBIA - As Boone County moves into the next phase of reopening, hair salons can now open their doors to... More >>
2 hours ago Thursday, May 28 2020 May 28, 2020 Thursday, May 28, 2020 6:17:00 PM CDT May 28, 2020 in News
COLUMBIA — As state and commercial testing is more available for the Missourians, the number of confirmed cases is rising.... More >>
6 hours ago Thursday, May 28 2020 May 28, 2020 Thursday, May 28, 2020 3:00:00 PM CDT May 28, 2020 in News
COLUMBIA - The City Manager of Columbia announced plans to further cut city budgets due to the effects of COVID-19.... More >>
6 hours ago Thursday, May 28 2020 May 28, 2020 Thursday, May 28, 2020 2:42:00 PM CDT May 28, 2020 in News
As COVID-19 spreads, KOMU 8 will continue to update you about the impacts on the community. You can find... More >>
6 hours ago Thursday, May 28 2020 May 28, 2020 Thursday, May 28, 2020 2:40:00 PM CDT May 28, 2020 in News
BOONE COUNTY - While other sports adjust the game plan to respect social distancing, some are just playing things business... More >>
6 hours ago Thursday, May 28 2020 May 28, 2020 Thursday, May 28, 2020 2:16:00 PM CDT May 28, 2020 in News
KANSAS CITY, Mo. (AP) — The University of Missouri-Kansas City says it will begin bringing back faculty, staff and students... More >>
6 hours ago Thursday, May 28 2020 May 28, 2020 Thursday, May 28, 2020 2:14:00 PM CDT May 28, 2020 in News
COLUMBIA — During the State of the City address, John Glascock said the city is leaving it up to the... More >>
7 hours ago Thursday, May 28 2020 May 28, 2020 Thursday, May 28, 2020 1:51:00 PM CDT May 28, 2020 in News
JEFFERSON CITY - Governor Mike Parson will deliver his daily COVID-19 briefing live from the State Capitol in Jefferson City... More >>
8 hours ago Thursday, May 28 2020 May 28, 2020 Thursday, May 28, 2020 12:46:00 PM CDT May 28, 2020 in News
Editor's note: Dr. Edith Bracho-Sanchez is a primary care pediatrician, director of pediatric telemedicine and assistant professor of pediatrics at... More >>
8 hours ago Thursday, May 28 2020 May 28, 2020 Thursday, May 28, 2020 12:26:23 PM CDT May 28, 2020 in A Brighter Tomorrow
COLUMBIA - Click below to watch the final candidate forum for the Columbia School Board. All four candidates - Jonathan... More >>
9 hours ago Thursday, May 28 2020 May 28, 2020 Thursday, May 28, 2020 11:30:00 AM CDT May 28, 2020 in News
INDEPENDENCE, Mo. (The Kansas City Star) - Police in suburban Kansas City say a domestic disturbance led to a shooting,... More >>
12 hours ago Thursday, May 28 2020 May 28, 2020 Thursday, May 28, 2020 8:47:00 AM CDT May 28, 2020 in News
(CNN) - President Donald Trump is set to announce an executive order against social media companies on Thursday, White House... More >>
13 hours ago Thursday, May 28 2020 May 28, 2020 Thursday, May 28, 2020 7:39:26 AM CDT May 28, 2020 in News
(CNN) - Hundreds remained on the streets of Minneapolis late Wednesday night protesting the death of George Floyd, a 46-year-old... More >>
15 hours ago Thursday, May 28 2020 May 28, 2020 Thursday, May 28, 2020 6:09:08 AM CDT May 28, 2020 in News
COLUMBIA — An employee at The Bluffs in Columbia tested positive for COVID 19, executive director Donna Bowers announced in... More >>
1 day ago Wednesday, May 27 2020 May 27, 2020 Wednesday, May 27, 2020 8:33:00 PM CDT May 27, 2020 in News
US— The fraught, freighted number of this particular American moment is a round one brimming with zeroes: 100,000. A hundred... More >>
1 day ago Wednesday, May 27 2020 May 27, 2020 Wednesday, May 27, 2020 8:31:00 PM CDT May 27, 2020 in News
COLUMBIA - The city of Columbia will not be collecting residential recycling curbside the week of May 25 after Columbia/Boone... More >>
1 day ago Wednesday, May 27 2020 May 27, 2020 Wednesday, May 27, 2020 6:33:00 PM CDT May 27, 2020 in News
FULTON - Three people were arrested on drug charges after a narcotics search occurred at 1504 Monroe Street in Fulton.... More >>
1 day ago Wednesday, May 27 2020 May 27, 2020 Wednesday, May 27, 2020 6:31:16 PM CDT May 27, 2020 in News
Columbia, MO
Broken Clouds 66°
10pm 63°
11pm 64°
12am 64°
1am 63°