Researchers are currently pursuing gene therapy as an area that may lead to a cure for Gaucher disease. It is believed that gene therapy will involve introducing normal glucocerebrosidase genes into cells of an affected individual. Ideally, these cells would then produce sufficient amounts of glucocerebrosidase. While research of gene therapy for Gaucher disease is ongoing, much work and time is needed before it may become available to the Gaucher community.
Lysosomal Storage Disorders
Gaucher disease is one of more than 40 rare inherited genetic disorders classified as a lysosomal storage disorder (LSD). Individually each LSD is relatively rare, but grouped together they affect about 1 in every 7,700 babies born. Learn more about Lysosomal Storage Disorders »
For more than two decades, the Gaucher Registry has been a global resource to the medical and patient communities, helping to improve outcomes in patients with Gaucher disease. Learn more about participating in the Gaucher Registry »