What are lysosomal storage disorders?
Lysosomal storage disorders (LSDs) are a group of rare inherited disorders, including Gaucher disease, in which there is abnormal storage of large chemical molecules in various organ systems in the body. Little data is available on the prevalence of LSDs, however, it is thought that one in 5-8,000 newborn babies may suffer from an LSD.
LSD in children and young people can cause deterioration in the function and structure of several body organs such as the brain, skeleton, internal organs such as the liver, kidneys, lungs, heart and spleen.
Some LSDs are treatable but there is currently no specific treatment available for the majority. However early diagnosis and treatment may prolong life and reduce disability in some LSDs.
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