Dustin Armstrong, PhD, president of Parasail, LLC, talks about the drug, VAL-1221, for the treatment of Pompe disease.  

Pompe disease is a lysosomal disorder that leads to reduced levels of acid alpha-glucosidase (GAA), which in turn leads to a buildup of glycogen in the cells. The accumulation of glycogen, especially in muscles, can lead to numerous complications.

As Dr. Armstrong explains in this video, VAL-1221 is a combination of enzyme replacement therapy with an antibody to help transport the enzyme to both lysosomal and extra-lysosomal storage of glycogen in cells. A phase I/II trial has begun and Dr. Armstrong is currently seeking funding to continue the development of this drug to treat Pompe disease.  

To stay informed about this and other lysosomal storage disorders, visit checkrare.com/diseases/lysosomal-storage-disorders/