Can Gene Therapy Transform the Lives of Patients with GSD1a?
April 6, 2023
April 6, 2024
Genetics, Hepatology, Endocrinology, Internal Medicine, Pediatricians, Primary Care
GSD1A, Glycogen Storage Disease type 1a, G6Pase Deficiency
This activity is jointly provided by Partners for Advancing Clinical Education (PACE) and PlatformQ Health Education, LLC.
In collaboration with the American Society of Gene + Cell Therapy (ASGCT).
This activity is supported by an independent educational grant from Ultragenyx.
- Physicians — maximum of 1.0 AMA PRA Category 1 Credit(s)™
All other healthcare professionals completing this course will be issued a statement of participation.
The educational design of this activity addresses the needs of geneticists, hepatologists, endocrinologists, internal medicine physicians, pediatricians, primary care physicians, NPs/PAs, nurses, and other HCPs involved in the care of patients living with GSD1a.
Statement of Need/Program Overview
For the rare genetic metabolic disorder, glycogen storage disease type 1a (GSD1a), liver transplantation is the only potential cure. Patients endure enormous physical and psychological challenges that significantly impact quality of life. New gene therapies in clinical trials have the potential to replace defective/missing enzymes via adenovirus vector serotype 8 (AAV8) and provide a much-needed treatment option for patients. Clinicians should understand these emerging therapies so they could be prepared to help patients with GSD1a take advantage of these options, when available.
You are invited to join us for an educational activity designed to review the efficacy and safety data on new therapeutic strategies for GSD1a, as well as the rationale for gene therapy and its ability to transcend the limitations of the standard-of-care. Led by a clinical geneticist and a hepatologist, “Can Gene Therapy Transform the Lives of Patients with GSD1a?”, will discuss the diagnosis and differentiation of GSD1a from other GSD types, review the limitations of dietary regulation and transplantation, and describe the mechanisms of action, benefits, and risks associated with emerging gene therapies. This activity will also help clinicians anticipate and mitigate treatment-related adverse events.
After completing this activity, the participant should be better able to:
- Describe the pathophysiology and long-term complications of GSD1a
- Discuss the diagnostic criteria for GSD1a and differentiation from other GSD types
- Recognize the potential of gene therapy to address limitations of the current standard-of-care for GSD1a
- Evaluate clinical data on emerging gene-therapy approaches to GSD1a management
David F. Rodriguez- Buritica, MD
Division of Genetics
Department of Pediatrics
McGovern Medical School
The University of Texas Health Science Center at Houston
Henry Lin, MD
Associate Professor of Pediatrics
Division of Gastroenterology
School of Medicine
Oregon Health & Science University
Joint Accreditation Statement
In support of improving patient care, this activity has been planned and implemented by Partners for Advancing Clinical Education (PACE) and PlatformQ Health Education, LLC. PACE is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.
Physician Credit Designation
PACE designates this enduring material for a maximum of 1.0 AMA PRA Category 1 Credit™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
Faculty and Disclosure of Conflicts of Interest
PACE requires instructors, planners, managers, and other individuals who are in a position to control the content of this activity to disclose all financial relationships they may have with ineligible companies. All relevant financial relationships are thoroughly vetted and mitigated according to PACE policy. PACE is committed to providing learners with high-quality accredited CE activities and related materials that promote improvements or quality in healthcare and not a specific proprietary business interest of an ineligible company.
The faculty reported the following relevant financial relationships or relationships to products or devices they have with ineligible companies related to the content of this educational activity:
- David F. Rodriguez- Buritica, MD
- Consultant, Advisor, Speaker: Biomarin, Inc.
- Researcher: Ultragenyx
- Henry Lin, MD
- Consultant, Advisor, Speaker: Albireo, Mirum
The PACE planners and others have no relevant financial relationship(s) to disclose with ineligible companies. The PlatformQ Health Education, LLC planners and others have no relevant financial relationship(s) to disclose with ineligible companies.
Instructions for Participation and Credit
In order to receive credit for this activity, the participant must (1) read the target audience, learning objectives, and disclosure statements, (2) complete the educational activity online, and (3) complete the post-test and activity evaluation. To receive AMA PRA Category 1 Credits™, participants must receive a score of 75% on the post-test.
There is no fee for this educational activity.
Course Viewing Requirements
Participants will need a computer with a recent version of Adobe Flash installed, as well as an internet connection sufficient for streaming media.
Disclosure of Unlabeled Use
This educational activity may contain discussion of published and/or investigational uses of agents that are not indicated by the FDA. The planners of this activity do not recommend the use of any agent outside of the labeled indications.
The opinions expressed in the educational activity are those of the faculty and do not necessarily represent the views of the planners. Please refer to the official prescribing information for each product for discussion of approved indications, contraindications, and warnings.
Participants have an implied responsibility to use the newly acquired information to enhance patient outcomes and their own professional development. The information presented in this activity is not meant to serve as a guideline for patient management. Any procedures, medications, or other courses of diagnosis or treatment discussed or suggested in this activity should not be used by clinicians without evaluation of their patient’s conditions and possible contraindications and/or dangers in use, review of any applicable manufacturer’s product information, and comparison with recommendations of other authorities.
Accreditation Support: For additional information about the accreditation of this activity, please visit https://partnersed.com.