Lysosomal Storage Diseases

Lysosomal Storage Diseases

What are Lysosomal Storage Diseases?

Lysosomal Storage Diseases are a group of about 50 disorders, characterized by a build-up of toxic materials in the body’s cells of the skeleton, skin, brain, heart and nervous system.  

Treatment of the symptoms depends on the specific Lysosomal Storage Disease the patient has but can include a bone marrow transplant, enzyme replacement therapy, and medication management.

Lysosomal Storage Diseases Doctors and Providers

Resources

More information is available at Climb National Information Centre for Metabolic Diseases (Email: ir.svcs@climb.org.uk) or through the Hide and Seek Foundation for Lysosomal Disease Research.