Enzyme Replacement Therapies

This activity is available for free to members of NASP. Visit the top section of www.ProCE.com/CSPE for details.


A product of the National Association of Specialty Pharmacy's (NASP) Center for Specialty Pharmacy Education(CSPE). View more NASP/CSPE Activities

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 leading to accumulation of waste products, impairment of various cellular metabolism such as sugar, lipid, proteins and nucleic acid, which result in complications of 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

The target audience for this activity includes pharmacists. Upon completion of this activity, participants will be able to:

  • Overview 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.


Lunawati Bennett, MS, MRE, PhD, PharmD, RPh, FACN
Professor of Pharmaceutical Sciences
College of Pharmacy
Union University
Jackson, Tennessee


It is the policy of ProCE, Inc. to ensure balance, independence, objectivity and scientific rigor in all of its continuing education activities. Faculty must disclose to participants the existence of any significant financial interest or any other relationship with the manufacturer of any commercial product(s) discussed in an educational presentation. Lunawati Bennett does not have any relevant commercial and/or financial relationships to disclose.

CE Accreditation

Release Date: June 1, 2020
Expiration Date: June 1, 2023


This CE activity is jointly provided by ProCE, Inc. and the National Association of Specialty Pharmacy (NASP). ProCE is accredited by the Accreditation Council for Pharmacy Education as a provider of continuing pharmacy education. ACPE Universal Activity Number 0221-9999-20-223-H01-P has been assigned to this knowledge-based, home-study activity. This CE activity is approved for 1.25 contact hour (0.125 CEU) in states that recognize ACPE providers. Completion of an evaluation and post-test with a score of 70% or higher is required to receive CE credit. Proof of completion will be posted in NABP CPE Monitor profiles. No partial credit will be given.


No external support has been received for this activity.


The material presented in this CE activity does not reflect the views of ProCE, Inc. or the commercial sponsor. These materials may discuss uses and dosages for therapeutic products, processes, procedures and inferred diagnoses that have not been approved by the United States Food and Drug Administration. A qualified health care professional should be consulted before using any therapeutic product discussed. All readers and continuing education participants should verify all information and data before treating patients or employing any therapies described in this continuing education activity.