Delandistrogene Moxeparvovec

Description

Delandistrogene moxeparvovec is a gene therapy developed for the treatment of Duchenne muscular dystrophy (DMD), a rare genetic disorder caused by mutations in the DMD gene that prevent the production of dystrophin, a protein essential for muscle strength and function.

This therapy uses an adeno-associated virus (AAV) vector to deliver a shortened but functional form of the dystrophin gene—called micro-dystrophin—directly into muscle cells. By enabling production of this micro-dystrophin protein, Elevidys aims to slow disease progression and improve muscle performance in affected patients.