mobility

Most people love the beach. Whether it’s your first time going to the beach or not, seaside vacations, and traveling in general, are almost always enjoyable. But what if you can’t get to the ocean once you’re there? Disabled individuals must find other ways…

Walking distance improved in late-onset Pompe disease patients on enzyme replacement therapy (ERT), while their lung function and muscle strength remained unchanged, a meta-analysis has found. More studies are, however, needed to confirm these findings and “to obtain more…

Pompe disease is characterized by progressive muscle weakness and disability. A number of adaptive devices are available to maintain patients’ quality of life. What is Pompe disease? Pompe disease is a rare genetic disorder caused by mutations in the GAA gene. This gene contains the instructions necessary…

Treatment with Myozyme (alglucosidase alfa, marketed as Lumizyme in the U.S.) improves walking ability and may stabilize respiratory function in adults with Pompe disease during the first three years of treatment, according to a recent study. However, factors such as aging may explain the loss of benefits observed at later stages. The…

While Pompe disease can affect the heart, respiratory system, and other functions, its chief symptom is muscle weakness. Exercise and physical therapy can help patients preserve mobility for as long as possible. Following are answers to some frequently asked questions about Pompe disease and exercise. How does…

Avalglucosidase alfa, an investigational next-generation enzyme replacement therapy (ERT), leads to clinically meaningful improvements in respiratory muscle strength and mobility in children and adults with late-onset Pompe disease (LOPD), according to top-line data from a Phase 3 trial. The findings will be the foundation for applications that…

When used in combination with enzyme replacement therapy, albuterol improves lung and motor function in patients with late-onset Pompe disease (LOPD), a Phase 1/2 trial showed. Trial findings were reported in the study, “Improved muscle function in a phase I/II clinical trial of albuterol…

Wearable technology that quantifies daily physical activity, such as FitBit One, can help determine mobility patterns and evaluate the clinical status of people with late-onset Pompe disease, a study shows. The study, “Mobility assessment using wearable technology in patients with late-onset Pompe disease,” was published…

A neuron-specific gene therapy treatment improved motor coordination in a mouse model of Pompe disease and highlights the role that neuronal dysfunction plays in this disease. The study, “A Neuron-Specific Gene Therapy Relieves Motor Deficits in Pompe Disease Mice,” was published in the journal Molecular…