Clenbuterol as an add-on therapy appears to be safe and significantly improves the effectiveness of enzyme replacement therapy (ERT) in patients with late-onset Pompe disease, according to the results from a Phase 1/2 trial. The study, “Correction of Biochemical Abnormalities and Improved Muscle Function in a Phase…
enzyme replacement therapy
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…