Novartis's gene therapy, Zolgensma, is already approved in the U.S. for children under the age of two with spinal muscular atrophy (SMA), a severe genetic disorder that affects the muscles and is the leading genetic cause of infant mortality. Now, the treatment has the potential to expand its use to older children, following promising results from a late-stage trial. The trial focused on patients aged 2 to less than 18 who had never received prior treatment and were unable to

Gene therapy has shown promise in treating many genetic diseases, but some conditions, like muscular dystrophy, involve very large genes that are too big to be delivered using current methods. A new technology called StitchR has solved this problem and could offer a treatment option for these hard-to-treat conditions. Instead of trying to deliver the whole large gene at once, StitchR splits the gene into two smaller parts. These two parts are delivered separately into cells,