Duchenne disease treatment, dubbed “GNT0004”, follows the principles of gene therapy.
- To help people with Duchenne muscular dystrophy, researchers have developed a new treatment called “GNT0004”.
- “Children treated at the therapeutic dose improved their ability to walk, run, climb stairs and get up alone without or with support,” they say.
- Duchenne muscular dystrophy, or Duchenne muscular dystrophy, is a genetic disease that causes progressive degeneration of all the muscles in the body.
On the occasion of World Duchenne Muscular Dystrophy Day, Genethon takes stock of a new treatment currently being evaluated which can counteract the extremely serious effects of this disease.
Duchenne muscular dystrophy is one of the most common neuromuscular pathologies in children. “Nearly 250,000 people worldwide are affected by it and all are waiting for the treatment that will change their lives.” explains Généthon in a press release. “Gene therapy is today the most promising therapeutic avenue because it attacks the origin of the disease and not just its symptoms,” he adds.
“Good tolerance” of the new treatment for Duchenne muscular dystrophy
To follow this direction, Généthon has designed the “GNT0004”, a drug currently being evaluated in a trial conducted in France and the United Kingdom on patients with Duchenne muscular dystrophy.
“The results in the first patients presented at the Myology2024 congress in Paris last April show good tolerance of this drug candidate as well as efficacy data, both in terms of microdystrophin expression and functional improvement,” welcome the researchers who are members of the non-profit organization. “Indeed, children treated at the therapeutic dose improved their ability to walk, run, climb stairs and get up alone without or with support,” they indicate.
New treatment for Duchenne muscular dystrophy: “hope is now concrete”
On the strength of these initial results, Généthon is preparing, in conjunction with European Medicines Agency (EMA)the final phase of drug evaluation.
“After years of research during which Duchenne muscular dystrophy has taken so many lives, there is now concrete hope for us, families,” confides Laurence Tiennot-Herment, president of AFM-Telethon and mother of Charles-Henri, who died of Duchenne muscular dystrophy in 2003. “Seeing children who would inexorably lose their strength without treatment achieve such feats creates incredible emotion,” she describes. “We are not at the end of the road for this very complex and terrible disease, but never in our history have we obtained such results which confirm to us today that we are on the right track!” she rejoices.
Duchenne muscular dystrophy, or Duchenne muscular dystrophy, is a genetic disease that causes progressive degeneration of all the muscles in the body. “It is linked to an anomaly of the DMD gene, responsible for the production of a protein involved in supporting muscle fibers,” noted Inserm.
People with Duchenne muscular dystrophy have a life expectancy of between 30 and 40 years.