zocaglusagene nuzaparvovec clinical trials

Gene Transfer Study in Patients With Late Onset Pompe Disease

This study is looking into a new gene therapy called zocaglusagene nuzaparvovec (AT845) for adults with Late Onset Pompe Disease. Researchers want to find out if it's safe and if it helps improve the condition. It's an early-stage study, giving different doses to see which works best.