Low-dose treatment with methotrexate can reduce the immune reaction that often accompanies enzyme replacement therapies in infants with Pompe disease, researchers report. Their study, “An immune tolerance approach using transient low-dose methotrexate in the ERT-naïve setting of patients treated with a therapeutic protein: experience in infantile-onset Pompe…
enzyme replacement therapy
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…
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…
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