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Gene Replacement Therapy in Practice: Considerations & Case Studies in Process

Activity Snapshot

Activity Type: 
On-Demand Webinar
Release Date: 
Tuesday, November 21, 2023
Expiration Date: 
Wednesday, November 20, 2024


Bo Hoon Lee, MD
Assistant Professor, Department of Neurology
University of Rochester Medical Center
Natalie Goedeker, DNP, CPNP
Co-Director- Pediatric Neuromuscular Clinical Research Center
Washington University in St. Louis
Erin Collins, MS
Medication Access Manager
University of Rochester Medical Center
Activity Overview: 


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This Grand Rounds webinar with Bo Hoon Lee, MD and Erin Collins, Medication Access Manager, of the University of Rochester and Natalie Goedeker, CPNP, of Washington University in St. Louis who will discuss practicalities, infrastructural needs, and their firsthand learnings from experience administering Gene Replacement Therapy (GRT).

Learning Objectives: 

● Recognize and anticipate unique infrastructural needs for administering gene replacement therapies (GRT) for neuromuscular disease
● Identify specific opportunities for you and your care team to support patients receiving GRT
● Discuss how to locate resources to help integrate GRT into practice and/or support patients who receive GRT

Accreditation and Designation Statement: 

CE credit provided by AKH Inc., Advancing Knowledge in Healthcare.

In support of improving patient care, this activity has been planned and implemented by AKH Inc., Advancing Knowledge in Healthcare and MDA. AKH Inc., Advancing Knowledge in Healthcare 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. This activity was planned by and for the healthcare team, and learners will receive 1 Interprofessional Continuing Education (IPCE) credit for learning and change.

See full accreditation and designation statements and disclosure report here.

How to Obtain CME credits : 

To obtain a certificate of completion, a score of 70% or better on the post-test is required. Please proceed with the activity until you have successfully completed this program, answered all test questions, completed the post-test and evaluation, and have received a digital copy of your certificate. You must participate in the entire activity to receive credit. There is no fee to participate in this activity. If you have questions about this activity, please contact AKH Inc. at

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Faculty Disclosures: 

Name          Relationship      Commercial Interest

Erin Collins: N/A,  Nothing to disclose

Natalie Goedeker, DNP: Adviser; Sarepta

Bo Hoon Lee, MD: Researcher; AMO Pharma, Sarepta, Sanofi Genzyme

Bernadette Makar, MSN, APRN-BC, NP-C (planner/reviewer): N/A; Nothing to disclose

Dorothy Duffy, RPh (planner/reviewer): N/A; Nothing to disclose

AKH Inc Staff and Planners: N/A; Nothing to disclose

MDA Staff and Planners: N/A; Nothing to disclose

All of the relevant financial relationships listed for these individuals have been mitigated.


It is the policy of AKH Inc. to ensure independence, balance, objectivity, scientific rigor, and integrity in all of its continuing education activities. The author must disclose to the participants any significant relationships with ineligible companies whose products or devices may be mentioned in the activity or with the commercial supporter of this continuing education activity. Identified conflicts of interest are mitigated by AKH prior to accreditation of the activity. AKH planners and reviewers have no relevant financial relationships to disclose.

Disclosure of Unlabeled Use and Investigational Product

This educational activity may include discussion of uses of agents that are investigational and/or unapproved by the FDA. Please refer to the official prescribing information for each product for discussion of approved indications, contraindications, and warnings.


This course is designed solely to provide the healthcare professional with information to assist in his/her practice and professional development and is not to be considered a diagnostic tool to replace professional advice or treatment. The course serves as a general guide to the healthcare professional, and therefore, cannot be considered as giving legal, nursing, medical, or other professional advice in specific cases. AKH Inc. specifically disclaim responsibility for any adverse consequences resulting directly or indirectly from information in the course, for undetected error, or through participants misunderstanding of the content.