Employees of the Belgorod National Research University, together with the Artgen Biotech company, have developed a gene therapy drug that can potentially cure Mioshi muscular dystrophy. The drug has already passed preclinical trials.
Description. Miyoshi muscular dystrophy (MMD) is an autosomal recessive skeletal muscle disorder characterized by onset in young adulthood of distal muscle weakness affecting the upper and lower limbs but sparing the intrinsic hand muscles. This is a rare progressive disease associated with the absence of a protein responsible for the integrity of muscle cells. Today, there is no drug in the world for the treatment of Miyoshi dystrophy. In 2024, there are plans to submit an application to the Ministry of Health of the Russian Federation to seek permission to conduct a clinical trial of the drug in humans.