SBC-102

Information for Patients and Families:

LAL deficiency is a rare, progressive lysosomal storage disease (LSD) in which people do not have enough of the enzyme, lysosomal acid lipase. When there is not enough of this enzyme, or when it is missing, the body is not able to properly break down certain types of lipids (or fats) called cholesteryl esters and triglycerides. Significant health problems can result from this disease including fatty liver, cirrhosis (scarring) of the liver that can result in liver failure, and aggressive, early atherosclerosis (hardening of the arteries). Some people with LAL deficiency have milder symptoms while many others suffer premature death due to complications from the disease.

SBC-102 is being developed as a potential treatment for LAL deficiency. SBC-102 is a protein designed to be identical to the enzyme that is missing. It is being evaluated in preclinical studies as a potential enzyme replacement therapy, or ERT, which would replace the missing enzyme and help reduce the build-up of cholesteryl esters and triglycerides throughout the body.

Patients and Families: Click here if you would like to speak to someone at Synageva regarding LAL deficiency