CME Information

Activity Title

Highlights From ISTH 2021 Congress


Gene Therapy in Hemophilia

Accreditation Type

AMA PRA Category 1 Credit(s)™

Release Date

August 9, 2021

Expiration Date

August 8, 2022

Estimated Time to Complete Activity

30 minutes


Upon completion of the activity, participants should be able to:

David Lillicrap, MD, FRCPC
Queen’s University
Kingston, Ontario, Canada

Steven W. Pipe, MD
University of Michigan
Ann Arbor, Michigan

Wolfgang A. Miesbach, MD, PhD
Goethe University Hospital
Frankurt/Main, Germany

Margareth Ozelo, MD, PhD
University of Campinas
Campinas, São Paulo, Brazil


  1. There are no fees for participating in and receiving credit for this activity.
  2. Review the activity objectives and CME/CE information.
  3. Complete the CME/CE activity
  4. Complete the online posttest. A score of 75% is required to successfully complete this activity. The participant may take the test until successfully passed.
  5. Complete the CME/CE evaluation/attestation form, which provides each participant with the opportunity to comment on how participating in the activity will affect their professional practice; the quality of the instructional process; the perception of enhanced professional effectiveness; the perception of commercial bias; and his/her views on future educational needs.
  6. Credit documentation/reporting:
    • If you are requesting AMA PRA Category 1 Credits™ or a certificate of participation—your CME/CE certificate will be available for download.


This activity is provided by The France Foundation and produced in collaboration with the International Society on Thrombosis and Haemostasis.

This activity is intended for physicians (hematologists), nurse practitioners, physician assistants, nurses managing patients with hemophilia, and scientists with an interest in basic, translational, and clinical research in hemophilia around the world.

An estimated 400,000 people worldwide are living with hemophilia. A variety of increasingly effective treatment options have been available to patients over the past decades. However, globally, only 25% receive adequate treatment. After many years of research and clinical trials, gene therapy is moving towards its promise as a revolutionary new treatment option for patients with hemophilia. Significant progress has occurred over the last decade in gene therapy for patients with hemophilia. It is essential for all members of the hemophilia care team to be knowledgeable about gene therapy for hemophilia and poised for the integration of this new therapeutic approach into clinical practice.

This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of The France Foundation and the International Society on Thrombosis and Haemostasis. The France Foundation is accredited by the ACCME to provide continuing medical education for physicians.

The France Foundation designates this enduring activity for a maximum of 0.50 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

In accordance with the ACCME Standards for Commercial Support, The France Foundation (TFF) and the International Society on Thrombosis and Haemostasis (ISTH) require that individuals in a position to control the content of an educational activity disclose all relevant financial relationships with any commercial interest. TFF and ISTH resolve all conflicts of interest to ensure independence, objectivity, balance, and scientific rigor in all their educational programs. Furthermore, TFF and ISTH seek to verify that all scientific research referred to, reported, or used in a CME/CE activity conforms to the generally accepted standards of experimental design, data collection, and analysis. TFF and ISTH are committed to providing learners with high-quality CME/CE activities that promote improvements in health care and not those of a commercial interest.

Activity Staff Disclosures
The planners, reviewers, editors, staff, CME committee, or other members at The France Foundation who control content have no relevant financial relationships to disclose.

The planners, reviewers, editors, staff, CME committee, or other members at the ISTH who control content have no relevant financial relationships to disclose.

Faculty Disclosures–Activity Faculty
The following faculty report that they have relevant financial relationships to disclose:

The France Foundation (TFF) and the International Society on Thrombosis and Haemostasis (ISTH) require CME faculty (speakers) to disclose when products or procedures being discussed are off-label, unlabeled, experimental, and/or investigational, and any limitations on the information that is presented, such as data that are preliminary, or that represent ongoing research, interim analyses, and/or unsupported opinion. Faculty in this activity may discuss information about pharmaceutical agents that is outside of US Food and Drug Administration approved labeling. This information is intended solely for continuing medical education and is not intended to promote off-label use of these medications. TFF and ISTH do not recommend the use of any agent outside of the labeled indications. If you have questions, contact the Medical Affairs Department of the manufacturer for the most recent prescribing information.

TThis activity is supported by an independent medical educational grant from BioMarin, Freeline Therapeutics Limited, Pfizer Inc., Spark Therapeutics, and uniQure, Inc.

The France Foundation and the International Society on Thrombosis Haemostasis present this information for educational purposes only. The content is provided solely by faculty who have been selected because of recognized expertise in their field. Participants have the professional responsibility to ensure that products are prescribed and used appropriately on the basis of their own clinical judgment and accepted standards of care. The France Foundation, the International Society on Thrombosis Haemostasis, and the commercial supporter(s) assume no liability for the information herein.

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If you have questions about this CME activity, please contact The France Foundation at 860-434-1650 or