A viral vector engineered from a duck virus, called AAV.div3A, may offer a way to safely give patients more than…
Articles by Michela Luciano, PhD
Late-onset Pompe disease (LOPD), a rare, inherited muscle disorder, was finally diagnosed in a 52-year-old man in Austria after…
The buildup of the complex sugar glycogen, the main cause of muscle damage in late-onset Pompe disease (LOPD), can…
A simple, noninvasive breathing intervention that briefly exposes the body to repeated bouts of low oxygen modestly improved breathing in…
Using Nexviazyme (avalglucosidase alfa) led to sustained improvements in lung function, walking ability, and other motor skills over nearly…
Estimates of the number people diagnosed with Pompe disease vary widely across regions, with notably higher rates in populations…