The UNC technology, developed by Pediatrics Professor Chengwen Li, M.D., Ph.D., uses adeno-associated viruses (AAVs) to ferry ...

and Pompe disease result from waste buildup in cells, leading to serious health issues. Dysfunctions of the lysosomal recycling pathway are also found in Parkinson's and Alzheimer's diseases.

Pompe disease is a rare genetic disorder that can result in respiratory failure, a significant complication due to ...

The drug is a recombinant version of the enzyme alpha-glucosidase, which is deficient in Pompe disease, leading to the accumulation of glycogen to toxic levels that damage muscle cells.

The company’s most advanced programmes are in Pompe disease – a rare genetic disease causing buildup of a sugar molecule inside cells – Parkinson’s disease and congestive heart failure.

Glucose, the fuel used by the body’s cells for energy, is normally stored as glycogen in the liver and muscles. In Pompe disease, glycogen builds up in the cells of certain organs and muscles, ...

Megan’s affliction, Pompe disease, is exceedingly rare. The course of the disease varies by patient, but it causes children’s muscles to weaken and, eventually, their lungs may fail.

Aro Biotherapeutics has dosed the first patient in a Phase Ib trial of ABX1100, a novel treatment for late-onset Pompe disease (LOPD), a rare inherited disorder that causes progressive muscle ...