Experimental gene therapy for rare muscle disease shows early promise but study halted

This study tested a gene therapy called SRP-9003 for people with limb-girdle muscular dystrophy type 2E (LGMD2E), a rare genetic disease that causes muscle weakness. The treatment aimed to deliver a working gene to muscle cells to help them produce a missing protein. Only 6 people took part before the study was stopped early. The main goal was to check safety and see if the therapy could increase the protein levels in muscles.