Purpose: To review the narratives that detailed the most important features of Infantile Onset Pompe disease (IOPD) from the physician's perspective, submitted as part of a large multicenter, ...

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com. Back to Healio Alglucosidase alfa was today deemed safe ...

The primary manifestations of Pompe disease are muscle weakness and cardiomyopathy. Although accumulation of glycogen has also been seen in the nervous system in patients, the significance of brain ...

Researchers have successfully used gene therapy to treat patients with infantile onset Pompe disease, a progressive condition that severely compromises cardiopulmonary function in the first years of ...

The first test of in utero enzyme replacement therapy (ERT) proved safe and effective for a baby with infantile-onset Pompe disease, according to a case report. An infant with cross-reactive ...

RALEIGH, N.C., April 8, 2025 /PRNewswire/ -- GeneVentiv Therapeutics, a gene therapy company, today announced it has signed a global licensing agreement with Duke University for the first universal ...

Pompe disease is a rare genetic disorder that impacts both children and adults. It’s important to understand the next steps following a Pompe disease diagnosis to help prepare you and your loved ones ...

Pompe disease is caused by a deficiency in the lysosomal enzyme acid α-glucosidase (GAA) that leads to accumulation of glycogen in the lysosomes, mainly seen in skeletal and cardiac muscles.