lysosomal storage disorders
Lysosomal storage disorders are a group of inherited diseases caused by the malfunction of lysosomes, which are cellular structures responsible for breaking down waste materials and cellular debris. When lysosomes do not function properly, harmful substances can accumulate in cells, leading to various health problems. These disorders can affect multiple organs and systems in the body.
There are over 50 different types of lysosomal storage disorders, including Gaucher disease, Fabry disease, and Pompe disease. Symptoms can vary widely but often include developmental delays, organ enlargement, and neurological issues. Early diagnosis and treatment are crucial for managing these conditions effectively.