These therapies aim at preventing disease progression and thus improving quality of life. Examples of the use of enzyme replacement therapy are Mucopolysaccharidoses and Gaucher disease. Results have shown effectivity of enzyme replacement therapy. In some instances as malignant infantile osteopetrosis, hematopoietic stem cell transplantation can be life-saving. References


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Emotional disturbance may refer to:

Emotional and behavioral disorders, most frequently used in an educational context
Mental disorders, especially those involving emotions or emotional disorders
Emotional trauma
Emotional distress
Mania and depression
Anxiety, fear, extreme sadness or anger, and other emotions that may disturb the mental state of an individual