CESD: Late Onset Lysosomal Acid Lipase (LAL) Deficiency
LAL Deficiency occurs less frequently in infants than it does in children, adolescents or adults.  The early onset form of LAL Deficiency (Wolman Disease) strikes 1-2 babies for every million births and is rapidly fatal, usually in the first year.  Late onset LAL Deficiency (CESD) affects 25 individuals per million births and may lead to liver fibrosis, cirrhosis, liver failure and death. There also appears to be an increased risk of strokes because of potential build-up of lipid in the walls of major arteries (atherosclerosis). Learn more >

Wolman Disease: Early onset LAL Deficiency
Most people have never heard of Wolman Disease.  It is a very rare disease that affects only 1-2 babies out of every million and is rapidly fatal before the first year.  Wolman Disease is the early onset form of Lysosomal Acid Lipase (LAL) Deficiency and is a genetic condition.  As is the case for some other genetic disease (eg cystic fibrosis) parents may not know they are carriers of the abnormal gene until they have an affected child.  If both parents are carriers, there is a 1 in 4 chance that the baby will be born with Wolman Disease.  LAL Deficiency belongs to the family of diseased called Lysosomal Storage Disorders. Learn more >

Regulatory Process for the Approval of Drugs
Before any drug can be prescribed Regulatory agencies such as the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) require evidence that a drug is safe and effective.  Once this has been established a manufacturer receives a license to make the drug available for the general medical community’s use. Learn more >

Treatment Access
No treatments for LAL Deficiency have been studied in clinical trials, which means that there are no licensed medicines approved by the Regulatory authorities like the FDA.  As is the case for other diseases where there are no approved treatments, other treatments are used in an attempt to control or help  some of the signs and symptoms of the disease.  Learn more >

FAQ
Get answers to commonly asked questions about LAL Deficiency.  Learn more >