Russia To Develop Own Drug Treatment For Spinal Muscular Atrophy - FMBA

Russia to Develop Own Drug Treatment for Spinal Muscular Atrophy - FMBA

Russia's Center for Brain and Neurotechnologies of the Federal Biomedical Agency (FMBA) will launch a study to create its own drug treatment for spinal muscular atrophy, a rare genetic disease that causes weakness and wasting in muscles used for movement, FMBA head Veronika Skvortsova said on Wednesday

MOSCOW (UrduPoint News / Sputnik - 01st December, 2021) Russia's Center for Brain and Neurotechnologies of the Federal Biomedical Agency (FMBA) will launch a study to create its own drug treatment for spinal muscular atrophy, a rare genetic disease that causes weakness and wasting in muscles used for movement, FMBA head Veronika Skvortsova said on Wednesday.

"The Center's technologies allow to create such a drug, but it will be a long process, because it requires both validation and very complex clinical trials. But yes, there is such a possibility. And I know that Vsevolod Vadimovich (Belousov, director of the center) plans to launch such studies here," Skvortsova told reporters.

Spinal muscular atrophy affects the motor function of the body and causes the loss of motor neurons and progressive muscle wasting.

Children suffering from the disease can eventually stop moving and breathing. Without treatment, life expectancy in most severe cases does not exceed two years.

There are three drugs currently available for the treatment of the disease in the world, two of which have been registered in Russia Nusinersen (Spinraza) and Risdiplam (Evrysdi). One injection of Spinraza costs about $100,000 and it should be taken every four months for the entirety of the patient's life after the initial treatment stage, when it is injected every two weeks. A yearly course of Risdiplam reportedly costs about $340,000.

The third drug, Zolgensma, was named the most expensive drug in the world at $2.2 million, but only a single dose is required to swap the defective gene and treat the disease.

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