Cardiac function normalized within a year of starting enzyme replacement therapy (ERT) for children with classic infantile-onset Pompe…
Articles by Lindsey Shapiro, PhD
Late-onset Pompe disease (LOPD) patients exhibit significant gene activity changes in their skeletal muscles compared with healthy people, some…
Astellas Gene Therapies has obtained the rights to develop medication that could help reduce preexisting immune responses against the…
In 2022, Pompe Disease News brought you weekly coverage of the latest scientific research, clinical trial updates, and treatment advances…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to MZE001, Maze Therapeutics‘ investigational oral treatment…
A woman was diagnosed with advanced late-onset Pompe disease after three decades of being misdiagnosed, according to a recent…
Certain protein modifications may enhance the ability of a therapeutically-delivered acid alpha-glucosidase (GAA) enzyme to be secreted and taken up…
A small study in the U.K. is evaluating a noninvasive MRI-based tool that could potentially detect late-onset Pompe disease…
Administrating a stem cell gene therapy before beginning enzyme-replacement therapy (ERT) prevented the development of antibodies against the acid alpha-glucosidase…
Lower urinary tract symptoms, including a loss of bladder control and unusual urine flow, are prevalent among children with…