The year 2024 marked a significant milestone in the field of gene therapy for Duchenne muscular dystrophy (DMD) with the FDA approval of delandistrogene moxeparvovec. This AAV-based gene therapy, developed by Sarepta Therapeutics, delivers a synthetic micro-dystrophin transgene and has shown promise in slowing the progression of the disease. However, despite this progress, challenges remain in the field of gene therapy for DMD. Clinical trials conducted by various developers have encountered hurdles such as immune-mediated adverse events and early patient deaths, highlighting the complexities and risks associated with gene therapy in inherited disorders. While the approval of delandistrogene moxeparvovec represents a significant advancement, it also underscores the need for continued research and innovation to overcome the remaining obstacles in the pursuit of effective treatments for DMD and other inherited disorders.
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