Health
New “cocktail” medicines can benefit up to 45% of patients with Duchenne muscular dystrophy-ScienceDaily
A new “cocktail” drug being developed at the University of Alberta provides effective and economical treatment to relieve symptoms in up to 45% of patients with Duchenne muscular dystrophy (DMD), a chronic muscle-depleting disease. May be provided.
A team led by Toshifumi Yokota, a professor of medical genetics in the Faculty of Medicine and Dentistry, has created and is currently testing a cocktail of six treatments. This means that almost half of DMD patients can be treated with one. medicine.
DMD affects 6 out of 100,000 people, usually boys. People with DMD have various mutations in dystrophin, the body’s largest gene. Dystrophin is a protein that requires cells to remain intact. Dystrophin has 79 sections, or exons, and if any one is missing, the body cannot produce protein and the muscles degenerate.
There is no cure for DMD, but a new class of medicine uses an approach called “exon skipping.” It acts as a band-aid against lost exons, allowing the body to skip damaged instructions and produce the proteins needed to rebuild muscle tissue.
The US Food and Drug Administration has already approved other similar treatments, including Biltlasen, based on Yokota’s research. However, the scope of each is limited. This new “cocktail” treatment has the potential to help more patients.
“Each previously developed exon skipping molecule could treat only about 10% of DMD patients because of different mutations in exons at different locations within the gene,” said Yokota, a friend of Garrett Cumming. I am. Research & Muscular Dystrophy Canada Donated Research Chair.
“Our approach is to skip more than 11 exons at a time, which can treat about 45% of patients,” he explained.
Research this week Minutes of the National Academy of Sciences.
Yokota’s team tested a new synthetic drug in test tubes and muscle tissue from mouse patients. They found signs of dystrophin production, muscle building, and improved heart function.
DMD often leads to extreme skeletal weakness, but most patients actually die of heart failure. According to Yokota, existing exon skipping treatments do not penetrate the heart muscle. The limits this new cocktail deals with.
“Our cocktail is a combination of antisense oligonucleotides and new peptides that allow the drug to penetrate the heart muscle,” he said.
Cocktails still need to undergo toxicological tests and undergo regulatory procedures for conducting clinical trials. Yokota et al. Recently launched a company to support the commercialization of this drug.
Story source:
material Provided by University of Alberta.. Original written by Gillian Rutherford. Note: Content can be edited in style and length.
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