What is Lysosomal Storage Disease?

Lysosomal storage disease occurs when there is a deficiency of certain enzymes causing the lysosome in a cell to malfunction. This can lead to a number of disorders. Symptoms can vary depending on the disorder and they include seizures, blindness, deafness, and dementia.

Treatment for lysosomal storage disease includes taking medication to increase the needed enzymes. Common drug classes used for treatment include enzyme replacement therapies and GCS inhibitors.

Popular Lysosomal Storage Disease Drugs

Popularity Drug Name Drug Class Price
Enzyme Replacement Therapies 29,714 See Prices
Cystine Depleting Agents 138 See Prices
Enzyme Replacement Therapies 1,923 See Prices
see all 16 drugs

Note: Popularity is based on total prescriptions for the brand and generic versions of each drug, regardless of the condition being treated. Some drugs are prescribed for multiple conditions.

Lysosomal Storage Disease Drug Classes

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