Great progress has been made in the treatment of Duchenne disease, which is difficult to treat, in recent years; Two of the three types of new drugs are already available in Russia. In an interview about this “Izvestia” said Oleg Lapochkin, chief freelance pediatric neurologist of the Moscow region.
According to him, the first gene replacement therapy drug will be licensed in the summer of 2023.
“This is a completely new level of treatment: First, it is applied once by changing the affected part of the gene, and then it starts working as expected. As a result, the disease may not develop at all or may progress very slowly. The latter cause much more genetic “damage” than the former,” Lapochkin said.
The doctor explained that until recently the main method of treating progressive Duchenne muscular dystrophy was hormonal therapy, but its effect was unfortunately limited: the drug extended the life of patients by an average of two years, he said.
According to Lapochkin, gene replacement therapy may be indicated for approximately 50% of patients. Issues of access to it are now being actively discussed in Russia through the Kindness Foundation Foundation.
Progressive Duchenne muscular dystrophy is a serious inherited genetic disease that occurs primarily in boys. At the age of 8-10, patients stop walking, and by the age of 15, all kinds of serious complications develop.
On September 7, 2023, scientists created a microgeneTo treat Duchenne muscular dystrophy.
Russian scientists had previously figured out how to do it. restore Heart cells in diabetic patients.
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Source: Gazeta
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