David Kronn, MD, Associate Professor of Pediatrics at New York Medical College, describes the latest results from the mini-COMET phase II study testing avalglucosidase alfa to treat Pompe disease.

Avalglucosidase alfa is a second-generation enzyme replacement therapy being developed to treat patients with Pompe disease. It is engineered to increase receptor-mediated uptake and has a higher binding affinity than the currently approved enzyme replacement therapy (alglucosidase alfa).

Pompe disease is an inherited lysosomal disorder that leads reduced levels of acid alpha-glucosidase (GAA). The net result is a buildup of glycogen in the body’s cells. The accumulation of glycogen, especially in muscles, can lead to a plethora of problems.

There are 3 types of Pompe disease, classic infantile-onset, non-classic infantile-onset, and late-onset Pompe disease.

To learn more about Pompe disease, visit checkrare.com/pompe.

 

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