Health Minister Fahrettin Koca announced that with the approval of the SMA Scientific Committee, gene therapy will be applied to babies with SMA. In addition, it is aimed to develop the drug used in the treatment of SMA in Turkey.
A new treatment method is started for SMA patients, the number of which has increased recently in Turkey. For the treatment of rare diseases such as SMA, a hospital will be established in Istanbul for the application of cell and gene therapy by the Ministry of Health.
A definitive treatment method has not yet been discovered for SMA disease. The SMA Scientific Committee of the Ministry of Health said that the Zolgensma (gene therapy) method can be applied.
Making statements about the gene therapy method, Health Minister Fahrettin Koca said:
“There is a patient group where it is effective. We made our plan to be implemented. It will be implemented soon. There is also an important development in this regard. We are establishing a new hospital in Istanbul for the treatment of rare diseases. This hospital will be dedicated to the application of cell and gene therapies. There will also be a facility to produce drugs used in cell and gene therapy on the same campus as the hospital. In this way, we will produce the medicine ourselves and apply the treatment ourselves.”
What is gene therapy for SMA patients?
Gene therapy is applied to children younger than 2 years old. Although it does not have a 100% success rate, it shows significant improvement in the patient. The drug, called Zolgensma, replaces the missing or mutated SMN1 gene in SMA patients, increasing the amount of SMN in the patient. The price of the drug, which is administered at once via intravenous injection, is over 2 million dollars. While the incidence of SMA disease in the world is 1 in 10 thousand, the incidence in Turkey has been determined as 1 in 6 thousand.