What is the outlook for someone with a glycogen storage disease (GSD)?

The outlook depends on the type of GSD and the organs that are affected. Thanks to recent advancements in therapy, treatment is very effective in managing the types of glycogen storage disease that affect the liver. GSD type I was considered a fatal disorder until 1971, when it was discovered that providing a continuous source of glucose ameliorated the metabolic derangements associated with the disorder. In 1982, uncooked cornstarch began to be used as a continuous glucose source, and the prognosis for patients living with this disorder improved dramatically. People with these types can now live full, normal lives.

GSDs that are not treated properly can lead to problems such as liver failure, heart failure, and lung failure. Quality of life and expected length of life are decreased in these cases. Because they affect so many organ systems, GSD Type II (Pompe’s disease) and GSD Type IV (Andersen’s disease) are very hard to treat and can be fatal.

Research into enzyme replacement therapy and gene therapy is promising, which may improve the outlook for the future.

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