Treatment with enzyme replacement therapy can help children with classic infantile Pompe disease reach adulthood. However, it fails to prevent the long-term effects of the disease in the brain and consequent  development of intellectual disabilities, a study shows. The study titled “Classic infantile Pompe patients…
ERT
Duke Health researchers are planning to launch a Phase 1 trial to test the safety of a new investigational gene therapy they developed to treat patients with Pompe disease. They are currently screening (NCT03285126) adults with late-onset Pompe disease to select eligible participants for the upcoming trial.