In April 2020, for the first time, gene therapy of patients with spinal muscular atrophy (SMA) was carried out on the territory of the Russian Federation using the drug AVXS-101 (registered in the USA as Zolgensma®).

This means that in our country for the first time applied a gene (gene replacement) drug that affects the genetic nature of the disease.

To carry out the procedure, the Institute’s specialists underwent special training with the assistance of the drug manufacturer, AveXis (Novartis).

Currently, the AVXS-101 preparation is not registered for clinical use in Russia. Its import into RF was made according to vital indications for specific patients. In accordance with the requirements defined by federal law No. 61-ФЗ On the Circulation of Medicines, an appropriate decision of the medical commission of the medical institution was received for this, as well as approval for the import of the drug by the Russian Ministry of Health.

The organization of timely access to the drug was the result of joint efforts and the coordinated work of all involved parties - a medical institution, distributor and manufacturer of the drug. The implementation of this project would also be impossible without the support and assistance of the Ministry of Health of the Russian Federation. Thanks to this, patients with SMA were given the opportunity to undergo gene therapy without traveling to other countries. This is especially true today, during a period of extremely tense international epidemiological situation.