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► Metabolic (or lysosomal) storage diseases

Metabolic (or lysosomal) storage diseases

Also known as: lyosomal

Metabolic (or lysosomal) storage diseases

Metabolic storage diseases are a range of genetic diseases in which patients have insufficient or malfunctioning enzymes in cells. Enzymes help break down certain materials within the cells for proper cell function. Without an appropriate enzyme balance, materials within the cells can become toxic and accumulate in vital organs such as liver, brain and more.
 
Treatment: Treatments of metabolic storage disease can include physical therapy, surgery, medication, enzyme replacement therapy or a blood or marrow transplantation.


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