… following treatment with Elevidys, a gene therapy developed by Sarepta Therapeutics, the … compounding the risks associated with gene therapy. Sarepta has reported the incident … -rokl, is the only approved gene therapy for Duchenne muscular dystrophy (DMD …

Scientists from the Allen Institute and Seattle Children’s Research Institute have announced a breakthrough in the development of gene replacement therapies for Dravet syndrome, a rare form of epilepsy. According to details published in a Science …

… of this disease-modifying gene therapy.1 The findings were … are currently limited to non–gene therapy options like nusinersen (Spinraza; … of a one-time gene therapy would be especially coveted … the efficacy of the gene therapy into the intrathecal space …

… dystrophy (DMD) who received the gene therapy delandistrogene moxeparvovec (Elevidys; Sarepta) … that participants receiving the gene therapy experienced clinically meaningful and statistically … #47;view/dmd-gene-therapy-elevidys-fails-to-meet-primary …

… formulation of the company’s gene therapy Zolgensma for the treatment of … tested the one-time intrathecal gene therapy, coded OAV101 IT, in individuals … remarkable because the one-time gene therapy removes some of the burden …

… two-step approach to gene therapy The newly developed therapy … are commonly used in gene therapy. To address this, the … small viruses used in gene therapy to deliver genetic material … treated with the new gene therapy showed remarkable improvements. Seizures …

On Wednesday, Novartis AG NVS announced safety and efficacy results from the Phase 3 program for investigational intrathecal onasemnogene abeparvovec (OAV101 IT) in a broad population of patients aged two to <18 years with spinal muscular atrophy (SMA). …

… Medicine, suggested that despite early gene therapy, some patients may benefit from … necessity of supplemental DMTs following gene therapy. Although onasemnogene abeparvovec remains a … need for supplemental therapy after gene therapy in patients with spinal …