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Evolutions in Duchenne Muscular Dystrophy: Treatment Implications for the Present and Future

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  • Overview

    There are ongoing clinical trials and approvals for new genetic and non-genetic therapies for Duchenne muscular dystrophy. Determining the most suitable therapy for each patient is a developing process. This education covers dystrophin disruption and restoration, recent clinical trial outcomes from different treatment methods, and a case study exploring novel patient management strategies. 

  • Disclosure of Conflicts of Interest

    DISCLOSURE POLICY 

    In accordance with the ACCME Standards for Integrity and Independence in Accredited Continuing Education, The France Foundation (TFF) requires that individuals in a position to control the content of an educational activity disclose all relevant financial relationships with any commercial entity. TFF resolves all conflicts of interest to ensure independence, objectivity, balance, and scientific rigor in all its educational programs.  

    Furthermore, TFF seeks 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 is committed to providing learners with high-quality CME/CE activities that promote improvements in health care and not those of a commercial interest. 

    Planning committee members, faculty, reviewers, and activity staff have disclosed the following relevant financial relationships. All relevant financial relationships listed have been mitigated. 

     

    Name of Individual 

    Role in Activity  

    Name of Commercial Interest(s) 

    Nature of Relationship(s) 

    Mechanism(s) implemented to resolve conflict of interest  

    Aravindhan Veerapandiyan, MD 

    Planning Committee Member, Faculty 

    1. AMO Pharma, AveXis, Biogen, Catalyst, Novartis, Pfizer, PTC Therapeutics, Sarepta Therapeutics, Scholar Rock, UCB 

     

    1. AMO, Cure Duchenne, Fibrogen, Muscular Dystrophy Association, Parent Project Muscular Dystrophy, Pfizer, Octapharma, Regenxbio, Sarepta 

    1. Non-CE Consulting  

     

     

     

     

     

     

     

    2. Contract Research/Grant Support 

     

     

     

     

     

    All final planning decisions concerning content, learning objectives, and evaluation questions were made by the non-conflicted planning committee 

     

    Content was reviewed by a non-conflicted content reviewer to ensure that it is not commercially biased, is fair and balanced, and is based on scientific evidence and/or clinical reasoning 

    Crystal Proud, MD 

    Planning Committee Member, Faculty 

    1. Biogen, Genentech/Roche, Novartis Gene Therapies, Sarepta, Scholar Rock 

     

     

    1. Astellas, Biogen, CSL Behring, Fibrogen, Novartis Gene Therapies, Pfizer, PTC, Sarepta, Scholar Rock 

     

    1. Biogen 

     

    1. Non-CE Consulting/Advisory Bord 

     

     

     

     

     

     

    2. Contract Research 

     

     

     

     

     

     

    3. Non-CE Speakers Bureau 

    All final planning decisions concerning content, learning objectives, and evaluation questions were made by the non-conflicted planning committee 

     

    Content was reviewed by a non-conflicted content reviewer to ensure that it is not commercially biased, is fair and balanced, and is based on scientific evidence and/or clinical reasoning 

    Craig McDonald, MD 

    Planning Committee Member, Faculty 

    1. Avidity Biosciences, Capricor, Inc., Edgewise Therapeutics, Italfarmaco, PTC Therapeutics, Santhera Pharmaceuticals, Sarepta (Symbiotix), Sarepta Therapeutics 

     

    1. Non-CE Consulting 

     

     

     

     

     

     

     

    All final planning decisions concerning content, learning objectives, and evaluation questions were made by the non-conflicted planning committee 

     

    Content was reviewed by a non-conflicted content reviewer to ensure that it is not commercially biased, is fair and balanced, and is based on scientific evidence and/or clinical reasoning 

    Melissa Glasner, MS 

    Planning Committee member 

    No relevant financial disclosures 

    NA 

    NA 

    Heather Tarbox, MPH 

    TFF Staff 

    No relevant financial disclosures 

    N/A 

    N/A 

    Janis Vajdos, MS  

    TFF Staff 

    No relevant financial disclosures 

    N/A 

    N/A 

    Patrick Harty, PhD 

    TFF Content Reviewer 

    No relevant financial disclosures 

    NA 

    NA 

     

    Disclosure of Unlabeled Use: 

    TFF requires 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 are 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 does 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.  

  • Target Audience

    This educational activity is intended for healthcare professionals involved in the management of patients with Duchenne muscular dystrophy (DMD), including neurologists, neuromuscular specialists, and other members of the health care team who care for patients with DMD. 

  • Learning Objectives

    Upon completion of this activity, learners should be able to: 

    • Describe the role of dystrophin disruption and restoration in the progression and management of DMD 

    • Assess the latest clinical trial results across various treatment modalities for DMD 

    • Examine emerging approaches to DMD management that seek to align patient selection, treatment choice, and optimal initiation of therapy 

  • Accreditation and Credit Designation Statements

     In support of improving patient care, The France Foundation 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.  

    Physician Credit Designation 

    The France Foundation designatesthis enduring activity for a maximum of 1.0AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity. 

     

    Nurses 

    The France Foundation designates this activity for 1.0 contact hours. 

     

     

    Physician Associates

    The France Foundation has been authorized by the American Academy of PAs (AAPA) to award AAPA Category 1 CME credit for activities planned in accordance with AAPA CME Criteria. This activity is designated for 1.0 AAPA Category 1 CME credits. Approval is valid until April 3, 2025. PAs should only claim credit commensurate with the extent of their participation.  

     

    Pharmacists 

    This knowledge-based activity, UAN JA0007204-0000-24-051-H01-P, qualifies for 1.0 contact hours (0.10 CEUs) of continuing pharmacy education credit. Your CE credits will be electronically submitted to the NABP upon successful completion of the activity. Pharmacists with questions can contact NABP customer service (custserv@nabp.net). 

     

    All other healthcare professionals completing this course will be issued a statement of participation. 

  • Disclaimer

    The France Foundation presentsthis 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 and the commercial supporter(s) assume no liability for the information herein. 

    COPYRIGHT INFORMATION 

    Copyright © 2024 The France Foundation. Any unauthorized use of any materials on the site may violate copyright, trademark, and other laws. You may view, copy, and download information or software ("Materials") found on the Site subject to the following terms, conditions, and exceptions:  

    • The materials are to be used solely for personal, noncommercial, informational, and educational purposes. The materials are not to be modified. They are to be distributed in the format provided with the source clearly identified. The copyright information or other proprietary notices may not be removed, changed, or altered.  

    • Materials may not be published, uploaded, posted, or transmitted (other than as set forth herein) without The France Foundation's prior written permission 

    PRIVACY POLICY 

    The France Foundation protects the privacy of personal and other information regarding participants and educational collaborators. The France Foundation will not release personally identifiable information to a third party without the individual's consent, except when such information is required for reporting purposes to the ACCME. 
     
    The France Foundation maintains physical, electronic, and procedural safeguards that comply with federal regulations to protect against the loss, misuse, or alteration of information that we have collected from you. 
     
    Additional information regarding The France Foundation’s Privacy Policy can be viewed at http://www.francefoundation.com/privacy.  
     
    CONTACT INFORMATION 

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

     

  • Accredited Provider

    This activity is provided by The France Foundation.  

  • Commercial Support

    This activity is supported by an independent medical educational grant from Sarepta Therapeutics. 

  • System Requirements

    • Supported Browsers (2 most recent versions):
      • Google Chrome for Windows, Mac OS, iOS, and Android
      • Apple Safari for Mac OS and iOS
      • Mozilla Firefox for Windows, Mac OS, iOS, and Android
      • Microsoft Edge for Windows
    • Recommended Internet Speed: 5Mbps+

  • Publication Dates

    Release Date:

    Expiration Date:

Facebook Comments

Recommended
Details
Presenters
Related
Comments
  • Overview

    There are ongoing clinical trials and approvals for new genetic and non-genetic therapies for Duchenne muscular dystrophy. Determining the most suitable therapy for each patient is a developing process. This education covers dystrophin disruption and restoration, recent clinical trial outcomes from different treatment methods, and a case study exploring novel patient management strategies. 

  • Disclosure of Conflicts of Interest

    DISCLOSURE POLICY 

    In accordance with the ACCME Standards for Integrity and Independence in Accredited Continuing Education, The France Foundation (TFF) requires that individuals in a position to control the content of an educational activity disclose all relevant financial relationships with any commercial entity. TFF resolves all conflicts of interest to ensure independence, objectivity, balance, and scientific rigor in all its educational programs.  

    Furthermore, TFF seeks 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 is committed to providing learners with high-quality CME/CE activities that promote improvements in health care and not those of a commercial interest. 

    Planning committee members, faculty, reviewers, and activity staff have disclosed the following relevant financial relationships. All relevant financial relationships listed have been mitigated. 

     

    Name of Individual 

    Role in Activity  

    Name of Commercial Interest(s) 

    Nature of Relationship(s) 

    Mechanism(s) implemented to resolve conflict of interest  

    Aravindhan Veerapandiyan, MD 

    Planning Committee Member, Faculty 

    1. AMO Pharma, AveXis, Biogen, Catalyst, Novartis, Pfizer, PTC Therapeutics, Sarepta Therapeutics, Scholar Rock, UCB 

     

    1. AMO, Cure Duchenne, Fibrogen, Muscular Dystrophy Association, Parent Project Muscular Dystrophy, Pfizer, Octapharma, Regenxbio, Sarepta 

    1. Non-CE Consulting  

     

     

     

     

     

     

     

    2. Contract Research/Grant Support 

     

     

     

     

     

    All final planning decisions concerning content, learning objectives, and evaluation questions were made by the non-conflicted planning committee 

     

    Content was reviewed by a non-conflicted content reviewer to ensure that it is not commercially biased, is fair and balanced, and is based on scientific evidence and/or clinical reasoning 

    Crystal Proud, MD 

    Planning Committee Member, Faculty 

    1. Biogen, Genentech/Roche, Novartis Gene Therapies, Sarepta, Scholar Rock 

     

     

    1. Astellas, Biogen, CSL Behring, Fibrogen, Novartis Gene Therapies, Pfizer, PTC, Sarepta, Scholar Rock 

     

    1. Biogen 

     

    1. Non-CE Consulting/Advisory Bord 

     

     

     

     

     

     

    2. Contract Research 

     

     

     

     

     

     

    3. Non-CE Speakers Bureau 

    All final planning decisions concerning content, learning objectives, and evaluation questions were made by the non-conflicted planning committee 

     

    Content was reviewed by a non-conflicted content reviewer to ensure that it is not commercially biased, is fair and balanced, and is based on scientific evidence and/or clinical reasoning 

    Craig McDonald, MD 

    Planning Committee Member, Faculty 

    1. Avidity Biosciences, Capricor, Inc., Edgewise Therapeutics, Italfarmaco, PTC Therapeutics, Santhera Pharmaceuticals, Sarepta (Symbiotix), Sarepta Therapeutics 

     

    1. Non-CE Consulting 

     

     

     

     

     

     

     

    All final planning decisions concerning content, learning objectives, and evaluation questions were made by the non-conflicted planning committee 

     

    Content was reviewed by a non-conflicted content reviewer to ensure that it is not commercially biased, is fair and balanced, and is based on scientific evidence and/or clinical reasoning 

    Melissa Glasner, MS 

    Planning Committee member 

    No relevant financial disclosures 

    NA 

    NA 

    Heather Tarbox, MPH 

    TFF Staff 

    No relevant financial disclosures 

    N/A 

    N/A 

    Janis Vajdos, MS  

    TFF Staff 

    No relevant financial disclosures 

    N/A 

    N/A 

    Patrick Harty, PhD 

    TFF Content Reviewer 

    No relevant financial disclosures 

    NA 

    NA 

     

    Disclosure of Unlabeled Use: 

    TFF requires 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 are 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 does 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.  

  • Target Audience

    This educational activity is intended for healthcare professionals involved in the management of patients with Duchenne muscular dystrophy (DMD), including neurologists, neuromuscular specialists, and other members of the health care team who care for patients with DMD. 

  • Learning Objectives

    Upon completion of this activity, learners should be able to: 

    • Describe the role of dystrophin disruption and restoration in the progression and management of DMD 

    • Assess the latest clinical trial results across various treatment modalities for DMD 

    • Examine emerging approaches to DMD management that seek to align patient selection, treatment choice, and optimal initiation of therapy 

  • Accreditation and Credit Designation Statements

     In support of improving patient care, The France Foundation 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.  

    Physician Credit Designation 

    The France Foundation designatesthis enduring activity for a maximum of 1.0AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity. 

     

    Nurses 

    The France Foundation designates this activity for 1.0 contact hours. 

     

     

    Physician Associates

    The France Foundation has been authorized by the American Academy of PAs (AAPA) to award AAPA Category 1 CME credit for activities planned in accordance with AAPA CME Criteria. This activity is designated for 1.0 AAPA Category 1 CME credits. Approval is valid until April 3, 2025. PAs should only claim credit commensurate with the extent of their participation.  

     

    Pharmacists 

    This knowledge-based activity, UAN JA0007204-0000-24-051-H01-P, qualifies for 1.0 contact hours (0.10 CEUs) of continuing pharmacy education credit. Your CE credits will be electronically submitted to the NABP upon successful completion of the activity. Pharmacists with questions can contact NABP customer service (custserv@nabp.net). 

     

    All other healthcare professionals completing this course will be issued a statement of participation. 

  • Disclaimer

    The France Foundation presentsthis 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 and the commercial supporter(s) assume no liability for the information herein. 

    COPYRIGHT INFORMATION 

    Copyright © 2024 The France Foundation. Any unauthorized use of any materials on the site may violate copyright, trademark, and other laws. You may view, copy, and download information or software ("Materials") found on the Site subject to the following terms, conditions, and exceptions:  

    • The materials are to be used solely for personal, noncommercial, informational, and educational purposes. The materials are not to be modified. They are to be distributed in the format provided with the source clearly identified. The copyright information or other proprietary notices may not be removed, changed, or altered.  

    • Materials may not be published, uploaded, posted, or transmitted (other than as set forth herein) without The France Foundation's prior written permission 

    PRIVACY POLICY 

    The France Foundation protects the privacy of personal and other information regarding participants and educational collaborators. The France Foundation will not release personally identifiable information to a third party without the individual's consent, except when such information is required for reporting purposes to the ACCME. 
     
    The France Foundation maintains physical, electronic, and procedural safeguards that comply with federal regulations to protect against the loss, misuse, or alteration of information that we have collected from you. 
     
    Additional information regarding The France Foundation’s Privacy Policy can be viewed at http://www.francefoundation.com/privacy.  
     
    CONTACT INFORMATION 

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

     

  • Accredited Provider

    This activity is provided by The France Foundation.  

  • Commercial Support

    This activity is supported by an independent medical educational grant from Sarepta Therapeutics. 

  • System Requirements

    • Supported Browsers (2 most recent versions):
      • Google Chrome for Windows, Mac OS, iOS, and Android
      • Apple Safari for Mac OS and iOS
      • Mozilla Firefox for Windows, Mac OS, iOS, and Android
      • Microsoft Edge for Windows
    • Recommended Internet Speed: 5Mbps+

  • Publication Dates

    Release Date:

    Expiration Date:

Facebook Comments

Schedule30 May 2024