Neil Weinreb, MD, FACP provides a summary of research presented at WORLDSymposium 2020 focused on gene therapy to potentially treat Gaucher disease.

Gaucher disease is a rare lysosomal storage disorder in which glucocerebroside accumulates in cells and certain organs. The disorder is characterized by bruising, fatigue, anemia, low blood platelet count and enlargement of the liver and spleen. Numerous therapies are available to attenuate disease progression, but these treatments cannot cure the disease. A number of gene therapies are in development to remove the need for regular treatments.

This video compliments the CME activity entitled Gaucher Disease Highlights from WORLDSymposium 2020, which is supported by an educational grant from Genzyme Corporation.

To obtain credit for the CME/CE, please visit are learning center.