The Problem

If you are “normal” your body has the ability to clear your cells of waste, miss folded proteins, and other organelles.  If you have a lysosomal storage disease (LSD) you do not have this ability or it is severely reduced. The result is a buildup of waste, miss folded proteins, and so on. This is known as a buildup of the autophagic process. In normal cell function, the autophagic process aids the lysosome with its work.  So is the LSD the problem or the buildup of everything else? We believe the answer is both. The first paper listed below does a great job of explaining the issues. Once the problem has been established we intend on showing potential solutions to the problems that exist with LSDs.