Generium, in collaboration with Sirius University, has begun clinical trials of a drug to treat Duchenne muscular dystrophy. This disease affects children.
This is a project in the field of personalized medicine, where technologies become doctors. We are currently in the clinical research stage; this is gene therapy, and it is our original development
Duchenne muscular dystrophy is a rare genetic disease. It affects boys, although it sometimes occurs in girls. The main symptoms are muscle weakness and difficulty in movement. They begin to manifest in childhood and progress over time. The disease is not completely curable, but it is possible to slow its progression.
The scientists developing the drug emphasized that gene therapy is a highly effective method for treating severe diseases associated with impaired function of certain genes. A healthy copy of the gene is delivered to patients with a damaged gene structure using a virus.
Generium is a Russian innovative biotechnology company. It combines a world-class research institute, a center for managing preclinical and clinical research, and a high-tech production facility.
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