The Russian Ministry of Health has registered a drug for the treatment of spinal muscular atrophy “Zolgensma”. This was reported by RIA Novosti.
In Russia, two drugs were previously registered for the treatment of the disease – “Nusinersen” (“Spinraza”) and “Risdiplam”. One injection of Spinraza costs $ 125,000.
As a reminder, spinal muscular atrophy is a genetic disorder characterized by the development of progressive muscle weakness in young children. SMA is caused by a mutation in the SMN1 gene that disrupts motor neurons in the spinal cord.
One injection of the drug “Zolgensma”, replacing the “defective” gene with an artificial one, can heal a child and provide him with a normal life. However, one injection costs more than two million dollars, and it needs to be done urgently before the child grows up.
In 2020, there were 913 people in Russia with a diagnosis of SMA, of which 20 were children under two years of age. SMA is considered the leading cause of childhood death from genetic diseases in developed countries.
Source: Rosbalt
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