The first steps

The gene therapy with Elevidys can be started provided certain conditions are met. The child must be between four and five years old and they must be able to walk. Furthermore, they must not have antibodies against the tool used to deliver the gene to its destination. Duchenne disease is caused by genetic errors in the dystrophin gene that lead to the absence of the dystrophin protein. Without this protein, the muscles become weakened and damaged.

Elevidys delivers a shortened, functional form of the dystrophin protein into muscle cells. The treatment is a one-time administration in which a viral vector, a tool needed to deliver the gene to its destination, is used to get the medicine into the muscle cells.

And in our region?

The FDA's approval is based on the results of three clinical studies on the safety and efficacy of Elevidys. In Europe, they are waiting for the results of a fourth study before submitting an application to the European Medicines Agency.

Bottom line... there is a small glimmer of hope for children with Duchenne. Although it will still be a long time coming.