Astellas Gene Therapies

This study was designed to test a new gene therapy called ASP2016 for heart problems in people with Friedreich Ataxia, a rare genetic disease. The goal was to check safety and how well people tolerated the treatment, which involved a single infusion plus steroids to protect the t…

This study tests a one-time gene therapy called AT845 for adults with late-onset Pompe disease. The goal is to help the body produce the missing enzyme on its own, potentially reducing or replacing the need for regular enzyme infusions. The trial is small (11 participants) and fo…

This study tested a gene therapy called AT132 for children under 5 with a rare, serious muscle disease (XLMTM) that causes breathing problems. The goal was to see if the therapy could reduce the need for breathing machines. However, the study was stopped early because some childr…

This study looks at people with late-onset Pompe disease to measure antibodies that might interfere with future gene therapy. Participants are either new to enzyme replacement therapy or have been on it for at least six months. No treatment is given; instead, blood and urine samp…