efficacy

While the activity of the acid alpha-glucosidase (GAA) enzyme and glycogen buildup are hallmarks of Pompe disease, the lack of standardized assessments to quantify both parameters hampers evaluations of disease severity and treatment efficacy, according to a review study. All current methods, however, do confirm…

Switching from Lumizyme (alglucosidase alfa) to Nexviazyme (avalglucosidase alfa) was shown to improve breathing and motor function for most people with late-onset Pompe disease (LOPD) in a small real-world study. Many of the improvements, such as those seen in walking ability, were minimal, and did not reach…

Certain protein modifications may enhance the ability of a therapeutically-delivered acid alpha-glucosidase (GAA) enzyme to be secreted and taken up by neighboring cells in models of Pompe disease, a study suggested. Such approaches could offer ways to improve the effectiveness of gene therapies for Pompe, although more work is…

The use of enzyme replacement therapy is safe and effective in Japanese people living with Pompe disease, a new study shows. Titled “Long-Term Observation of the Safety and Effectiveness of Enzyme Replacement Therapy in Japanese Patients with Pompe Disease: Results From the Post-marketing Surveillance,” the study…

Results of a Phase 4 clinical trial support the effectiveness of Lumizyme (4,000 liters recombinant human GAA) for 52 weeks in maintaining clinical stability in Pompe disease patients previously treated with the therapy’s 160-liter formulation, called Myozyme. The study, “Efficacy, safety profile, and immunogenicity of alglucosidase alfa…