Enzyme Replacement Therapies Course

Enzyme Replacement Therapies (ERTs) Course is organized by ProCE, Inc.,,Release Date: June 1, 2020,Expiration Date: June 1, 2023,,Activity Description:,Enzyme replacement therapies (ERTs) play a crucial role in the treatment of lysosomal storage disease (LSDs), a group of rare, heterogenous disorders characterized by lysosomal dysfunction. Defects in genes encoding lysosomal proteins or enzymes lead to the accumulation of waste products, and impairment of various cellular metabolisms such as sugar, lipid, proteins, and nucleic acid, which result in complications for different organs. Three LSDs will be discussed including Gaucher disease, Anderson-Fabry disease, and Pompe disease. For each disease, biomarkers, symptoms, complications, and other therapeutic options besides ERTs will be addressed. Pharmacists may benefit from learning drug therapies for these rare diseases.,,Learning Objectives:,At the completion of this activity, the participant will be better able to:,• Overview of the common disease states managed by enzyme replacement therapy (ERT) products.,• Review medications utilized for the treatment.,• Review the management of infused ERT products, including product selection, adverse drug reactions (ADRs), and product administration (i.e. Filtration, dilution, rates).,• Discuss the advancement in oral therapy.,• Discuss investigational drugs in clinical trials.