real-world study

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,…

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…

Patients with classic infantile-onset Pompe disease could benefit from increased dosing of Myozyme (alglucosidase alfa), an enzyme replacement therapy (ERT) marketed as Lumizyme in the U.S., according to a real-world European study. Benefits were seen in improved survival and ability to walk. “On the basis of our results,…

In the first years of treatment, Myozyme (alglucosidase alfa) — marketed in the U.S. as Lumizyme — can effectively increase physical endurance, and improve lung function and muscle strength in adults with late-onset Pompe disease (LOPD), a real-world study found. Yet, despite this treatment, patients’ clinical outcomes, particularly lung…