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All relevant financial relationships for anyone with the ability to control the content of this educational activity are listed below and have been mitigated. Others involved in the planning of this activity have no relevant financial relationships.

Faculty

Francesco Muntoni, MD

Professor of Paediatric Neurology
Director of the Dubowitz Neuromuscular Centre
UCL Great Ormond Street Institute of Child Health
London, United Kingdom

Disclosures

Francesco Muntoni, MD, has the following relevant financial relationships:
Consultant or advisor for: Biogen; Novartis; PTC; Roche; Sarepta
Speaker or member of speakers bureau for: Biogen; Novartis; PTC; Roche; Sarepta
Research funding from: Biogen; Roche; Sarepta
Other: Board Membership: Dyne Therapeutics; Pfizer, Inc. Therapy development for: DMD

Benedikt Schoser, MD

Professor of Neurology
Friedrich-Baur-Institute
Department of Neurology
Ludwig-Maximilians-University of Munich
Munich, Germany

Disclosures

Benedikt Schoser, MD, has the following relevant financial relationships:
Consultant or advisor for: Amicus Therapeutics, Inc.; argenx; Astellas Pharma, Inc.; Bayer; Maze; Sanofi
Speaker or member of speakers bureau for: Alexion Pharmaceuticals, Inc.; Kedrion; Sanofi
Research funding from: Amicus Therapeutics, Inc.; Astellas Pharma, Inc.

Editor

Grace O’Malley, BSc, PhD

Associate Medical Education Director, WebMD Global, LLC

Disclosures

Grace O’Malley, BSc, PhD, has no relevant financial relationships.

Compliance Reviewer

Leigh Schmidt, MSN, RN, CNE, CHCP

Associate Director, Accreditation and Compliance, Medscape, LLC

Disclosures

Leigh Schmidt, MSN, RN, CNE, CHCP, has no relevant financial relationships.

Peer Reviewer

This activity has been peer reviewed and the reviewer has no relevant financial relationships.

CME

AAV-Based Gene Therapies at Our Doorstep: Understanding How They Work

Authors: Francesco Muntoni, MD; Benedikt Schoser, MD
CME Released: 4/26/2023
Valid for credit through: 4/26/2024, 11:59 PM EST

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Credits Available

Physicians - maximum of 0.75 AMA PRA Category 1 Credit(s)™

You Are Eligible For
- Letter of Completion

Target Audience and Goal Statement

This educational activity is intended for an international audience of non-US pediatric neurologists, neurologists, neuromuscular disease specialists, pediatricians, and primary care physicians.

The goal of this activity is for learners to be better able to explain gene therapy, its expectations, and how it is implemented, highlighting the role of AAV vectors.

Upon completion of this activity, participants will:

Have increased knowledge regarding the
- Advantages of using AAV-based vectors for gene therapy
- Mechanisms by which AAV vectors enable cellular transduction and express a functional transgene
- Potential risks associated with AAV-mediated gene therapy

Disclosures

Faculty

Francesco Muntoni, MD

Professor of Paediatric Neurology
Director of the Dubowitz Neuromuscular Centre
UCL Great Ormond Street Institute of Child Health
London, United Kingdom

Disclosures

Francesco Muntoni, MD, has the following relevant financial relationships:
Consultant or advisor for: Biogen; Novartis; PTC; Roche; Sarepta
Speaker or member of speakers bureau for: Biogen; Novartis; PTC; Roche; Sarepta
Research funding from: Biogen; Roche; Sarepta
Other: Board Membership: Dyne Therapeutics; Pfizer, Inc. Therapy development for: DMD

Benedikt Schoser, MD

Professor of Neurology
Friedrich-Baur-Institute
Department of Neurology
Ludwig-Maximilians-University of Munich
Munich, Germany

Disclosures

Benedikt Schoser, MD, has the following relevant financial relationships:
Consultant or advisor for: Amicus Therapeutics, Inc.; argenx; Astellas Pharma, Inc.; Bayer; Maze; Sanofi
Speaker or member of speakers bureau for: Alexion Pharmaceuticals, Inc.; Kedrion; Sanofi
Research funding from: Amicus Therapeutics, Inc.; Astellas Pharma, Inc.

Editor

Grace O’Malley, BSc, PhD

Associate Medical Education Director, WebMD Global, LLC

Disclosures

Grace O’Malley, BSc, PhD, has no relevant financial relationships.

Compliance Reviewer

Leigh Schmidt, MSN, RN, CNE, CHCP

Associate Director, Accreditation and Compliance, Medscape, LLC

Disclosures

Leigh Schmidt, MSN, RN, CNE, CHCP, has no relevant financial relationships.

Peer Reviewer

This activity has been peer reviewed and the reviewer has no relevant financial relationships.

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For Physicians

Medscape, LLC designates this enduring material for a maximum of 0.75 AMA PRA Category 1 Credit(s)™ . Physicians should claim only the credit commensurate with the extent of their participation in the activity.

The European Union of Medical Specialists (UEMS)-European Accreditation Council for Continuing Medical Education (EACCME) has an agreement of mutual recognition of continuing medical education (CME) credit with the American Medical Association (AMA). European physicians interested in converting AMA PRA Category 1 Credit™ into European CME credit (ECMEC) should contact the UEMS (www.uems.eu).

College of Family Physicians of Canada Mainpro+® participants may claim certified credits for any AMA PRA Category 1 credit(s)™, up to a maximum of 50 credits per five-year cycle. Any additional credits are eligible as non-certified credits. College of Family Physicians of Canada (CFPC) members must log into Mainpro+® to claim this activity.

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For questions regarding the content of this activity, contact the accredited provider for this CME/CE activity noted above. For technical assistance, contact [email protected]

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There are no fees for participating in or receiving credit for this online educational activity. For information on applicability and acceptance of continuing education credit for this activity, please consult your professional licensing board.

This activity is designed to be completed within the time designated on the title page; physicians should claim only those credits that reflect the time actually spent in the activity. To successfully earn credit, participants must complete the activity online during the valid credit period that is noted on the title page. To receive AMA PRA Category 1 Credit™, you must receive a minimum score of 75% on the post-test.

Follow these steps to earn CME/CE credit*:

Read about the target audience, learning objectives, and author disclosures.
Study the educational content online or print it out.
Online, choose the best answer to each test question. To receive a certificate, you must receive a passing score as designated at the top of the test. We encourage you to complete the Activity Evaluation to provide feedback for future programming.

You may now view or print the certificate from your CME/CE Tracker. You may print the certificate, but you cannot alter it. Credits will be tallied in your CME/CE Tracker and archived for 6 years; at any point within this time period, you can print out the tally as well as the certificates from the CME/CE Tracker.

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From Medscape Education

CME

AAV-Based Gene Therapies at Our Doorstep: Understanding How They Work

Authors: Francesco Muntoni, MD; Benedikt Schoser, MDFaculty and Disclosures

CME Released: 4/26/2023

Valid for credit through: 4/26/2024, 11:59 PM EST

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Disclaimer

The educational activity presented above may involve simulated, case-based scenarios. The patients depicted in these scenarios are fictitious and no association with any actual patient, whether living or deceased, is intended or should be inferred.

The material presented here does not necessarily reflect the views of Medscape, LLC, or any individuals or commercial entities that support companies that support educational programming on medscape.org. These materials may include discussion of therapeutic products that have not been approved by the US Food and Drug Administration, off-label uses of approved products, or data that were presented in abstract form. These data should be considered preliminary until published in a peer-reviewed journal. Readers should verify all information and data before treating patients or employing any therapies described in this or any educational activity. A qualified healthcare professional should be consulted before using any therapeutic product discussed herein.

Faculty and Disclosures

Faculty

Francesco Muntoni, MD

Benedikt Schoser, MD

Editor

Grace O’Malley, BSc, PhD

Compliance Reviewer

Leigh Schmidt, MSN, RN, CNE, CHCP

Peer Reviewer

CME

AAV-Based Gene Therapies at Our Doorstep: Understanding How They Work

Credits Available

You Are Eligible For

Target Audience and Goal Statement

Disclosures

Faculty

Francesco Muntoni, MD

Benedikt Schoser, MD

Editor

Grace O’Malley, BSc, PhD

Compliance Reviewer

Leigh Schmidt, MSN, RN, CNE, CHCP

Peer Reviewer

Accreditation Statements

For Physicians

Instructions for Participation and Credit

AAV-Based Gene Therapies at Our Doorstep: Understanding How They Work

Learning Feedback Questions

PDF Downloads

Transcript Downloads