What Are Lysosomal Storage Disorders?

Lysosomal Storage Diseases are a group of about 50 disorders, which are characterized by an abnormal build up of various toxic materials in the body’s cells caused by enzyme deficiencies. They affect different parts of the body including the skeleton, skin, brain, heart and nervous system.

 

Treatment

Treatment of the symptoms depends on the specific Lysosomal Storage Disease the patient has but can include – Bone marrow transplant, enzyme replacement therapy, the drug imiglucerase (Cerezyme), the drug Aldurazyme.

 

Voluntary Sector Organisations