About the Lysosomal Disease Network

Although individually rare “orphan” conditions, the lysosomal diseases collectively affect 1 in 6,000 individuals and are responsible for a significant disability and disease burden. These diseases have become a test-bed for some of the most innovative and advanced experimental treatments, including treatment agents designed to cross the blood-brain barrier.

The rarity of each lysosomal disease means that no single medical research center has an opportunity to see the entire spectrum, or to acquire sufficient patient numbers to adequately test new therapies. The combined and integrated efforts of the NIH-funded Lysosomal Disease Network focus limited resources into a network of centers with expertise in one or more of these diseases in order to solve major challenges in diagnosis, disease management, and therapy. Solutions to these problems will have direct impact on patients suffering from lysosomal diseases, and important implications for medical practice.


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