On April 5, 2022, a scientific symposium “SMA gene therapy: the experience of Kazakhstan” was held at the National Scientific Center for Motherhood and Childhood of the University Medical Center Corporate Foundation.
In our country, this topic is now being actively discussed and arouses great public interest. The reason for this was the information that an expensive drug, onasemogen abeparvovek, was purchased with the financing of the Public Fund “Kazakhstan Khalkyna”. This drug was purchased for children with a diagnosis of spinal muscular atrophy, related to rare (orphan) diseases.
The Ministry of Health of the Republic of Kazakhstan has identified the National Research Center for Motherhood and Childhood of the UMC as the clinical base for the administration of the drug onasemogen abeparvovek. And to date, the drug has been used in 3 children at the Center. The fourth child is scheduled for April 7, 2022. Doctors assess the condition of sick children as satisfactory.
The chief freelance neurologist of the Ministry of Health of the Republic of Kazakhstan, Professor Altynshash Dzhaksybayeva, at a meeting attended by specialists of the Ministry of Health of the Republic of Kazakhstan, the public Foundation “Kazakhstan Khalkyna”, the NAO “Astana Medical University”, the NUO “Kazakh-Russian Medical University” and others, told about the algorithm, control plan and experience of using gene therapy in Kazakhstan.
Spinal muscular atrophy is a genetic disease that affects the motor neurons of the spinal cord and leads to muscle weakness. This is one of the most common causes of infant mortality from hereditary diseases. For a long time it was considered an incurable disease. However, in December 2016, the first drug entered the pharmaceutical market. In the same year, it was approved for use in the USA, in 2017 in the European Union and in 2019 in Russia.
Currently, 128 children have been diagnosed with such a diagnosis in Kazakhstan. In the future, their number may reach up to 250. Accordingly, experts stressed the importance of treating those who need help, using all the possibilities of state and public organizations.
Speaking at the scientific symposium, the Chairman of the Orphan Diseases Committee of the public foundation “Kazakhstan Khalkyna” Bakhyt Baigalieva thanked the specialists of the National Scientific Center for Motherhood and Childhood of the UMC CF for the successful treatments and expressed confidence that in the future joint activities aimed at preserving the health and life of the population of the country will also be carried out.
Press service of CF “UMC”