Posted by Michael Wonder on 20 Jun 2024
FDA expands approval of gene therapy for patients with Duchenne muscular systrophy
20 June 2024 - Today, the FDA expanded the approval of Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for the treatment of Duchenne muscular dystrophy for ambulatory and non-ambulatory individuals 4 years of age and older with Duchenne muscular dystrophy with a confirmed mutation in the Duchenne muscular dystrophy gene.
Elevidys was previously approved under accelerated approval for ambulatory individuals 4 through 5 years of age with Duchenne muscular dystrophy with a confirmed mutation in the Duchenne muscular dystrophy gene.
