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CPD

The Future of Duchenne Muscular Dystrophy: Understanding the Science Behind Emerging Gene Therapies

  • Authors: Crystal M. Proud, MD; Eugenio Mercuri, MD, PhD; Laurent Servais, MD, PhD
  • CPD Released: 5/18/2022
  • Valid for credit through: 5/18/2023
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  • Credits Available

    Physicians - maximum of 0.50 AMA PRA Category 1 Credit(s)™

    You Are Eligible For

    • Letter of Completion

Target Audience and Goal Statement

This activity is intended for an international audience of non-US neurologists, pediatricians, and other healthcare professionals caring for patients with DMD. 

The goal of this activity is that learners will be better able to understand the rationale for gene therapy in DMD and the latest data.

Upon completion of this activity, participants will:

  • Have increased knowledge regarding the
    • Science of gene therapy in DMD 
    • Current approaches being investigated for gene therapy in DMD 
    • Clinical data on emerging gene therapies for DMD


Disclosures

WebMD Global requires every individual in a position to control educational content to disclose all financial relationships with ineligible companies that have occurred within the past 24 months. Ineligible companies are organizations whose primary business is producing, marketing, selling, re-selling, or distributing healthcare products used by or on patients.

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 with ineligible companies.

Disclosures for additional planners can be found here.


Faculty

  • Crystal M. Proud, MD

    Director of Neurology and Neuromuscular Medicine
    Children's Hospital of The King's Daughters
    Norfolk, Virginia
    United States

    Disclosures

    Disclosure: Crystal M. Proud, MD, has the following relevant financial relationships:
    Consultant or advisor for: AveXis/Novartis Gene Therapies; Biogen; Genentech/Roche; Sarepta; Scholar Rock
    Speaker or member of speakers bureau for: Biogen
    Contracted researcher for: AveXis/Novartis Gene Therapies; Biogen; CSL Behring; Fibrogen; Pfizer; PTC; Sarepta; Scholar Rock

  • Eugenio Mercuri, MD, PhD

    Professor of Pediatric Neurology
    Catholic University
    Rome, Italy

    Disclosures

    Disclosure: Eugenio Mercuri, MD, PhD, has the following relevant financial relationships:
    Consultant or advisor for: Biogen; Italfarmaco; Novartis; Pfizer; PTC; Roche; Sarepta; Scholar Rock
    Speaker or member of speakers bureau for: Biogen; Novartis; PTC; Roche; Sarepta
    Research funding from: Biogen

  • Laurent Servais, MD, PhD

    Professor of Pediatric Neuromuscular Diseases
    MDUK Oxford Neuromuscular Centre
    University of Oxford
    United Kingdom

    Disclosures

    Disclosure: Laurent Servais, MD, PhD, has the following relevant financial relationships:
    Consultant or advisor for: Affinia; Audentes; Biohaven; Biogen; Catabasis; Dynacure; Evox Therapeutics; Novartis; Roche; RegenexBio; Sarepta; Scholar Rock
    Speaker or member of speakers bureau for: Audentes; Biogen; Novartis; Roche
    Research funding from: Biogen; Novartis; Roche

Editor

  • Katherine Carpenter

    Medical Education Director, WebMD Global, LLC

    Disclosures

    Katherine Carpenter, PhD has the following relevant financial relationship:
    Advisor or consultant for: Eisai, GW Pharmaceuticals

  • Eloise Ballard, PhD

    Scientific Content Manager, WebMD Global, LLC

    Disclosures

    Disclosure: Eloise Ballard, PhD, has no relevant financial relationships.

Compliance Reviewer

  • Leigh Schmidt, MSN, RN, CMSRN, CNE, CHCP

    Associate Director, Accreditation and Compliance, Medscape, LLC

    Disclosures

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

Peer Reviewer

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


Accreditation Statements

    For Physicians

  • The Faculty of Pharmaceutical Medicine of the Royal Colleges of Physicians of the United Kingdom (FPM) has reviewed and approved the content of this educational activity and allocated it 0.50 continuing professional development credits (CPD).

    Contact WebMD Global

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]


Instructions for Participation and Credit

There are no fees for participating in or receiving credit for this online educational activity. For information about your eligibility to claim credit, 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 participating in the activity. To successfully earn credit, participants must complete the activity online during the credit eligibility period that is noted on the title page.

Follow these steps to claim a credit certificate for completing this activity:

  1. Read the information provided on the title page regarding the target audience, learning objectives, and author disclosures, read and study the activity content and then complete the post-test questions. If you earn a passing score on the post-test and we have determined based on your registration profile that you may be eligible to claim CPD credit for completing this activity, we will issue you a CPD credit certificate.
  2. Once your CPD credit certificate has been issued, you may view and print the certificate from your CME/CE Tracker. CPD 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 by accessing "Edit Your Profile" at the top of the Medscape Education homepage.

We encourage you to complete an 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 by accessing "Edit Your Profile" at the top of your Medscape homepage.

*The credit that you receive is based on your user profile.

CPD

The Future of Duchenne Muscular Dystrophy: Understanding the Science Behind Emerging Gene Therapies

Authors: Crystal M. Proud, MD; Eugenio Mercuri, MD, PhD; Laurent Servais, MD, PhDFaculty and Disclosures

CPD Released: 5/18/2022

Valid for credit through: 5/18/2023

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Contents of This CPD Activity

All sections of this activity are required for credit.

The Future of Duchenne Muscular Dystrophy: Understanding the Science Behind Emerging Gene Therapies

Dr Proud introduces DMD, its natural history, and treatment approaches.
Chrystal M. Proud, MD

The Science of Gene Therapy

Dr Servais discusses the principles of gene therapy and the challenges of applying this approach to Duchenne.
Laurent Servais, MD, PhD

The Evolving Therapeutic Landscape of Duchenne Muscular Dystrophy

Dr Mercuri discusses the various gene therapy approaches currently under development in DMD.
Eugenio Mercuri, MD, PhD

Clinical Data on Emerging Gene Therapies for Duchenne Muscular Dystrophy

Dr Proud discusses the latest clinical trial data for gene therapies currently in development in DMD.
Chrystal M. Proud, MD

Summary and Key Takeaways

Dr Proud summarizes the program and provides some key takeaways.
Chrystal M. Proud, MD
 

Educational Impact Challenge

Note: It is the policy of Medscape Education to avoid the mention of brand names or specific manufacturers in accredited educational activities. However, manufacturer names related to gene therapy candidates are provided in this activity in an effort to promote clarity. The use of manufacturer names should not be viewed as an endorsement by Medscape of any specific product or manufacturer.

The goal of this activity is that learners will be better able to understand the rationale for gene therapy in DMD and the latest data.

Before you begin this activity, please assess your clinical knowledge by completing this brief survey. Answering these questions again after the activity will allow you to see what you learned and to compare your answers with those of your peers.

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