Tag Archives: gene

FDA Approves Zolgensma (onasemnogene abeparvovec-xioi) Gene Therapy to Treat Pediatric Patients with Spinal Muscular Atrophy

FDA Approves Zolgensma (onasemnogene abeparvovec-xioi) Gene Therapy to Treat Pediatric Patients with Spinal Muscular Atrophy Print this page May 24, 2019 — The U.S. Food and Drug Administration today approved Zolgensma (onasemnogene abeparvovec-xioi), the first gene therapy approved to treat children less than two years of age with spinal muscular atrophy (SMA), the most severe… Read More »

Gene therapy stocks plunge after poor trial data

(Reuters) – Shares of U.S. gene therapy companies sank on Thursday after drugs developed by two small firms failed to show promise in separate clinical trials, underscoring the challenges in an emerging, lucrative field of biotech. Experimental treatments developed by Sangamo Therapeutics Inc and Solid BioSciences did not show enough clinical benefit to patients in… Read More »