walking abilities

Small changes in walking ability can be meaningful in advanced LOPD

For most adults with late-onset Pompe disease (LOPD), a fairly significant improvement in walking ability is needed to be noticeable, but for patients who are already experiencing difficulty walking, a slowing of decline can be meaningful, a new study reports. “Patients with severe baseline conditions should not be expected…

MDA 2023: Data support AT-GAA’s long-term benefits for LOPD patients

After two years being treated with AT-GAA (cipaglucosidase alfa/miglustat), adults with late-onset Pompe disease (LOPD) maintained their improvements in walking ability and had stabilized breathing function, as well as a reduction in the levels of disease biomarkers, according to…

#MDA2022 – AT-GAA Improves Walking, Lung Function, 3-year Data Show

Treatment with the two-part investigational therapy AT-GAA improved walking ability and lung function for up to three years among adults with Pompe disease in a Phase 1/2 clinical trial. Barry Byrne, MD, PhD, from the University of Florida, presented the findings at the 2022 MDA Clinical & Scientific Conference,…

Myozyme Effectiveness in LOPD Fades Over Time, Real-world Study Finds

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…