Lysosomal Storage Diseases
Lysosomal storage diseases are genetic disorders in which lysosomes – the cell organelles responsible for digesting cell waste and foreign particles – fail to function properly. This results in a build-up of certain materials in the body's cells, leading to a range of clinical symptoms including developmental delay, neurological problems, organ dysfunction, or failure. These diseases are usually progressive and may be life-threatening if not treated. Fortunately, many of these diseases can be treated with enzyme replacement therapy, which involves supplementing a patient's own missing enzymes with synthetic or animal-derived enzymes. This treatment is often successful in reducing or eliminating symptoms and can even lead to long-term improvements in quality of life.
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