Gene Therapy for Hemophilia: What Does the Future Hold?
December 22, 2022
December 22, 2023
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This activity is jointly provided by Physicians’ Education Resource®, LLC and CGTlive™
This activity is supported by an educational grant from CSL Behring LLC.
- Physicians — maximum of 0.75 AMA PRA Category 1 Credits™
All other healthcare professionals completing this course will be issued a statement of participation.
This educational activity is directed toward hematologist/oncologists, pediatricians, emergency medicine clinicians, geneticists, genetic counselors, nurse practitioners, physician assistants, registered nurses, and other health care professionals involved in the management of hemophilia.
Despite recent improvements in options for its treatment, hemophilia continues to have a significant negative impact on patients’ functionality and health-related quality of life. Gene therapy for hemophilia has the potential to reduce the treatment burden for patients and their care providers by eliminating the need for regular prophylaxis. Gene therapy for hemophilia presents a challenge to the existing structure of hemophilia treatment centers and requires enhanced collaboration and information-sharing to maximize benefits and minimize risks.
During this Clinical Consultations™, 2 expert faculty will discuss the role of gene therapy in treating patients with hemophilia. They will discuss the research that has led to the selection of viral vectors, the results of clinical trials to date, and the changes that need to occur within the health care system and hemophilia treatment centers to accommodate the use of this exciting new therapy.
- Upon completion of this activity, participants should be better able to:
- Assess the role of gene therapy in the treatment of hemophilia A and B
- Analyze clinical trial findings regarding gene therapy in patients with hemophilia A and B
Wolfgang Miesbach, MD
Director, Department of Blood Coagulation and Comprehensive Haemophilia Centre Medical Clinic
University Hospital Frankfurt
Steven W. Pipe, MD
Laurence A. Boxer Research Professor of Pediatrics and Communicable Diseases Professor of Pathology
Director, Pediatric Hemophilia and Coagulation Disorders Program
Medical Director, Special Coagulation Laboratory
University of Michigan
Ann Arbor, MI
Physicians’ Education Resource®, LLC, is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.
Physicians’ Education Resource®, LLC, designates this enduring material for a maximum of 0.75 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
Faculty, Staff, and Planners’ Disclosures
The staff of Physicians’ Education Resource®, LLC have no relevant financial relationships with ineligible entities.
- Wolfgang Miesbach, MD
- Grant/Research Support: Bayer, Biotest, CSL Behring, LFB, Novo Nordisk, Octapharma, Pfizer, Takeda/Shire
- Consultant: Bayer, Biomarin, Biotest, Chugai, CSL Behring, Freeline, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, Sanofi, Takeda/Shire, uniQure
- Speakers’ Bureau: Bayer, Biomarin, Biotest, Chugai, CSL Behring, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, Takeda/Shire
- Steven W. Pipe, MD
- Consultant: ApcinteX, ASC Therapeutics, Bayer, Biomarin, CSL Behring, Freeline Therapeutics, GeneVentiv, HEMA Biologics, Novo Nordisk, Pfizer, Regeneron/Intellia, Roche/Genentech, Sanofi, Spark Therapeutics, Takeda, uniQure
PER® mitigated all COI for faculty, staff, and planners prior to the start of this activity by using a multistep process.
Instructions for Participation and Credit
- Complete the activity (including pre- and post-activity assessments).
- Answer the evaluation questions.
- Request credit using the drop-down menu.
Course Viewing Requirements
Internet Explorer 8.0+ for Windows 2003, Vista, XP, Windows 7, Windows 8.1 and above
Google Chrome 28.0+ for Windows, Mac OS, or Linux
Mozilla Firefox 23.0+ for Windows, Mac OS, or Linux
Safari 6.0+ for Mac OSX 10.7 and above
Supported Phones & Tablets:
Android 4.0.3 and above
iPhone/iPad with iOS 6.1 or above
Off-Label Disclosure and Disclaimer
This activity may or may not discuss investigational, unapproved, or off-label use of drugs. Learners are advised to consult prescribing information for any products discussed. The information provided in this accredited activity is for continuing education purposes only and is not meant to substitute for the independent clinical judgment of a health care professional relative to diagnostic, treatment, or management options for a specific patient’s medical condition. The opinions expressed in the content are solely those of the individual faculty members and do not reflect those of PER® or any company that provided commercial support for this activity.