Varia Maiier Needs Your Help!

Until recently, there was no specific treatment for SLC6A1. However, thanks to the development of gene therapy, scientists have created a method that allows a healthy copy of the gene to be “delivered” to the body’s cells. Clinical trials are currently underway, and Varia has a chance to be part of this program or receive treatment immediately after its approval.

The development and production of a gene therapy drug is an extremely complex and expensive process. Each dose is manufactured individually in specialized laboratories in the USA. The cost also includes medical support, rehabilitation, and necessary examinations before and after the drug’s administration.

Gene therapy is a modern and globally recognized method for treating genetic diseases. It is not an “experiment” in the common sense, but a high-tech medical treatment developed over years by the world’s leading geneticists. Similar methods are already being successfully used to treat other rare diseases, such as SMA.

The therapy aims to eliminate the cause of the disease, not just its symptoms. Introducing a healthy gene allows the body to start producing the necessary protein, which stops the disease’s progression and allows the brain to develop correctly. This can significantly reduce or completely stop seizures and improve cognitive functions.