In patients with Pompe disease, treatment with Myozyme (alglucosidase alfa) is most often administered in outpatient settings and is rarely associated with adverse reactions. That’s according to real-world data spanning a little more than seven years in France. The treatment is marketed as Lumizyme in the U.S. Overall,…
Enzyme replacement therapy (ERT) for Pompe disease can be safely implemented at home, according to a study in the Netherlands. “Insights from this study can be used as a base for implementing home-based ERT in other countries and to further optimize patient care,” researchers wrote. The study, “Home-based…
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…
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