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Lysosomal Storage Disorders
Abstract
You are reading a NORD Rare Disease Report Abstract. NORD’s full collection of reports on over 1200 rare diseases is available to subscribers (click here for details). We are now also offering full Rare Disease reports to visitors who register on our website, for free. If you have already registered or if you are a subscriber, clicking the link to view the full report will give you an opportunity to log in. You will also be able to register or to reset your password. Registration is free and we do not share your information with anyone else, but you are limited to two full reports a day.
NORD is very grateful to Joe T.R. Clarke, MD, PhD, Director of the Genetic Metabolic Disease Program at The Hospital for Sick Children in Toronto for assistance in the preparation of this report.
Synonyms of Lysosomal Storage Disorders
- No synonyms found.
Disorder Subdivisions
- No subdivisions found.
General Discussion
Lysosomal storage diseases are inherited metabolic diseases that are characterized by an abnormal build-up of various toxic materials in the body's cells as a result of enzyme deficiencies. There are nearly 50 of these disorders altogether, and they may affect different parts of the body, including the skeleton, brain, skin, heart, and central nervous system. New lysosomal storage disorders continue to be identified. While clinical trials are in progress on possible treatments for some of these diseases, there is currently no approved treatment for many lysosomal storage diseases.
Organizations related to Lysosomal Storage Disorders
NORD provides referrals to many patient organizations dealing with specific lysosomal storage diseases. Please see our reports on the specific topics for those organizations. The following organizations offer general information on lysosomal storage diseases.
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